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Viral and nonviral delivery systems for gene delivery
Nouri Nayerossadat1, Talebi Maedeh2, Palizban Abas Ali3
1 Molecular Genetic Laboratory, Alzahra Hospital; Pediatric Inherited Disease Research Center, Isfahan University of Medical Sciences, Isfahan, Iran
2 Molecular Genetic Laboratory, Alzahra Hospital, Isfahan University of Medical Sciences, Isfahan, Iran
3 Department of Clinical Biochemistry, Faculty of Pharmacy and Pharmaceutical Sciences Research Center, Isfahan University of Medical Sciences and Health Services, Isfahan, Iran
| Date of Submission | 13-Dec-2011 |
| Date of Acceptance | 10-Mar-2012 |
| Date of Web Publication | 06-Jul-2012 |
Correspondence Address:
Nouri Nayerossadat
Molecular Genetic Laboratory, Alzahra Hospital, Isfahan University of Medical Sciences, Isfahan
Iran
 Source of Support: None, Conflict of Interest: None  | Check |
DOI: 10.4103/2277-9175.98152
Gene therapy is the process of introducing foreign genomic materials into host cells to elicit a therapeutic benefit. Although initially the main focus of gene therapy was on special genetic disorders, now diverse diseases with different patterns of inheritance and acquired diseases are targets of gene therapy. There are 2 major categories of gene therapy, including germline gene therapy and somatic gene therapy. Although germline gene therapy may have great potential, because it is currently ethically forbidden, it cannot be used; however, to date human gene therapy has been limited to somatic cells. Although numerous viral and nonviral gene delivery systems have been developed in the last 3 decades, no delivery system has been designed that can be applied in gene therapy of all kinds of cell types in vitro and in vivo with no limitation and side effects. In this review we explain about the history of gene therapy, all types of gene delivery systems for germline (nuclei, egg cells, embryonic stem cells, pronuclear, microinjection, sperm cells) and somatic cells by viral [retroviral, adenoviral, adeno association, helper-dependent adenoviral systems, hybrid adenoviral systems, herpes simplex, pox virus, lentivirus, Epstein-Barr virus)] and nonviral systems (physical: Naked DNA, DNA bombardant, electroporation, hydrodynamic, ultrasound, magnetofection) and (chemical: Cationic lipids, different cationic polymers, lipid polymers). In addition to the above-mentioned, advantages, disadvantages, and practical use of each system are discussed. Keywords: Chemical delivery, gene therapy, non viral delivery systems, physical delivery, viral delivery systems
How to cite this article:
Nayerossadat N, Maedeh T, Ali PA. Viral and nonviral delivery systems for gene delivery. Adv Biomed Res 2012;1:27 |
| Introduction | |  |
Basically gene therapy is an intracellular delivery of genomic materials (transgene) into specific cells to generate a therapeutic effect by correcting an existing abnormality or providing the cells with a new function. [1] Different types of gene delivery systems may be applied in gene therapy to restore a specific gene function or turning off a special gene(s). The ultimate goal of gene therapy is single administration of an appropriate material to replace a defective or missing gene. [2] The first human gene transfer was utilized in 1989 on tumor-infiltrating lymphocytes [3],[4] and the first gene therapy was done on ADA gene for treatment of patients with SCID (Severe Combined Immunodeficiency Defect) in 1990. [5] Although initially the main focus of gene therapy was on inherited genetic disorders, now diverse diseases, including autosomal or X-linked recessive single gene disorders (CF(Cystic Fibrosis), ADA (Adenosine Deaminase) -SCID, emphysema, retinitis pigmentosa, sickle cell anemia, phenylketonuria, hemophilia, DMD (Duchenne Muscular Dystrophy), some autosomal dominant disorders, even polygenic disorders, different forms of cancers, vascular disease, neurodegenerative disorders, inflammatory conditions, and other acquired diseases are targets of gene therapy. To date, thousands of disorders have been treated by more than hundreds of protocols of gene therapy. [1] There are 2 major categories of gene therapy: Germline gene therapy and somatic gene therapy. Although germline gene therapy may have a great potential, because it is currently ethically forbidden, it cannot be used. [6],[7],[8] To date, human gene therapy has been limited to somatic cell alterations and there is a remarkable development in the field. There are different viral and nonviral vectors for gene delivery, but all gene therapy applications depend on the fact that the genetic material needs to be delivered across the cell membrane and ultimately to the cell nucleus. Each of the delivery systems has some advantages and disadvantages, and in this review we explain about all types of gene delivery systems briefly [Figure 1].
| Different Methods of Gene Therapy | | |
Germline gene therapy
The technology of this type of gene therapy is simple as genetic abnormalities can be corrected by direct manipulation of germline cells with no targeting, and not only achieve a cure for the individual treated, but some gametes could also carry the corrected genotype. Although it almost never has been tested on humans, some different transgenic techniques have been used on other species, which include the following:
- Gene delivery to the nuclei taken from somatic cells at metaphase stage. [9],[10]
- Ex vivo alteration of egg cells, following in vitro fertilization. [11],[12]
- Manipulation of embryonic stem cells of mouse during in vitro culture by different gene delivery systems. [12],[13],[14]
- Pronuclear microinjection of exogenous DNA solution by a glass needle. [15]
- Transgenic delivery into sperm cells by direct or indirect injection to testis or other parts of the genital system. [16],[17]
Somatic gene therapy
Somatic gene therapy involves the insertion of genes into diploid cells of an individual where the genetic material is not passed on to its progeny. Somatic cell therapy is viewed as a more conservative, safer approach because it affects only the targeted cells in the patient, and is not passed on to future generations; however, somatic cell therapy is short-lived because the cells of most tissues ultimately die and are replaced by new cells. In addition, transporting the gene to the target cells or tissue is also problematic. Regardless of these difficulties, however, somatic cell gene therapy is appropriate and acceptable for many disorders.
There are 3 types of somatic gene therapy
Ex vivo delivery
In this system the genetic material is explanted from the target tissue or bone marrow, cultivated and manipulated in vitro, and then transducted and/or transfected into the target tissue. There are no immunologic problems in this way but only the technique is used in cases where the target cells act as protein secretion resources (like the treatment of ADA or hemophilia) or as a vaccine for cancer treatment, so there are major limitations on the use of ex vivo delivery. In addition, at present only a small percentage of reimplanted cells remain viable. [18],[19]
In situ delivery
The administration of the genetic material directly into the target tissue is in situ delivery. As most of the current delivery systems need no effective targeting, the way is proper. The system has been utilized in the delivery of CFTR gene by lipid and adenoviral vectors to a specific site in the respiratory tract and is also used in the treatment of different cancers. However, low efficiency of transduction is the main problem of this system, because in cancer therapy one malignant cell can re-establish the tumor again. [20],[21],[22]
In vivo delivery
The transfer of genetic material through an appropriate vector, which can be a viral or nonviral vector, into the target tissue is in vivo delivery. This technique is the least advanced strategy at present but potentially it might be the most useful. The problem of this way is insufficient targeting of vectors to the correct tissue sites; however, improvement in targeting and vector development will solve the problem.
| Different Vector Systems for Gene Delivery | | |
Viral vectors
One of the successful gene therapy systems available today are viral vectors, such as retrovirus, adenovirus (types 2 and 5), adeno-associated virus, herpes virus, pox virus, human foamy virus (HFV), and lentivirus. [23] All viral vector genomes have been modified by deleting some areas of their genomes so that their replication becomes deranged and it makes them more safe, but the system has some problems, such as their marked immunogenicity that causes induction of inflammatory system leading to degeneration of transducted tissue; and toxin production, including mortality, the insertional mutagenesis; and their limitation in transgenic capacity size. [24],[25] During the past few years some viral vectors with specific receptors have been designed that could transfer the transgenes to some other specific cells, which are not their natural target cells (retargeting). [26]
Retroviral vectors
Retroviral vectors are one of the most frequently employed forms of gene delivery in somatic and germline gene therapies. Retroviruses in contrast to adenoviral and lentiviral vectors, can transfect dividing cells because they can pass through the nuclear pores of mitotic cells; this character of retroviruses make them proper candidates for in situ treatment. [27],[28] In addition, all of the viral genes have been removed, creating approximately 8 kb of space for transgenic incorporation. Retroviruses are useful for ex vivo delivery of somatic cells because of their ability to linearly integrate into host cell genome; for example, they have been used for human gene therapy of X-SCID successfully but incidence of leukemia in some patients occurred because of integration of retroviruses to the LMO2 gene and inappropriate activation of it. [29],[30],[31],[32],[33],[34] Retroviral vectors also have been applied for familial hyperlipidemia gene therapy and tumor vaccination. However, the main limitations of retroviral vectors are their low efficiency in vivo, immunogenic problems, the inability to transduce the nondividing cells and the risk of insertion, which could possibly cause oncogene activation or tumor-suppressor gene inactivation. [27],[28],[29],[30],[31],[32],[33],[34]
Adenoviral vectors
Adenoviral vectors have been isolated from a large number of different species, and more than 100 different serotypes have been reported. Most adults have been exposed to the adenovirus serotypes most commonly used in gene therapy (types 2 and 5). Adenoviruses type 2 and 5 can be utilized for transferring both dividing and nondividing cells and have low host specificity so can be used for gene delivery into large range of tissues. [35] Adenoviruses are able to deliver large DNA particles (up to 38 kb), [36] but in contrast to retroviruses, as they would not integrate into the host genome, their gene expression is too short term. Natural and acute immunologic responses against adenoviruses have made their clinical application limited to a few tissues, such as liver, lung (especially for CF(Cystic Fibrosis) treatment), or localized cancer gene therapy. Although the risk of serious disease following natural adenovirus infection is rare and the viral genome would not integrate into the host genome, gene therapy by adenoviral vectors has caused serious bad side effects and even death of some patients. [37],[38],[39],[40] Recently, in addition to safety of these vectors, several essential genes have been deleted so that viral replication can only occur under control and also most of the viral genome is deleted to obtain sufficient space for 38 kb of transgene particles, this kind of adenoviruses are called "gutless" or "pseudo" adenoviruses.
Adeno-associated vectors
Adeno-associated vectors (AAV) are like adenoviral vectors in their features but because of having some deficiency in their replication and pathogenicity, are safer than adenoviral vectors. [41] In human, AAVs are not associated with any disease. Another special character of AAV is their ability to integrate into a specific site on chromosome 19 with no noticeable effects cause long-term expression in vivo. The major disadvantages of these vectors are complicated process of vector production and the limited transgene capacity of the particles (up to 4.8 kb). AAVs have been used in the treatment of some diseases, such as CF, hemophilia B, Leber congenital amaurosis, and AAT (Alpha-1 antitrypsine) deficiency. [41],[42],[43],[44]
Helper-dependent adenoviral vector
Helper-dependent adenoviral vector (HdAd), called also as "gutless" or "gutted" vector, are last generation of adenovirus vectors. [35] The disadvantages of the first-generation AdV, such as a packaging capacity limitation (8 kb), immunogenicity, and toxicity, could be overcome, with the development of high-capacity "gutless" Advs (HC-AdV). In this helper-dependent vector system, one vector (the helper) contains all the viral genes required for replication but has a conditional gene defect in the packaging domain. The second vector contains only the ends of the viral genome, therapeutic gene sequences, and the normal packaging recognition signal, which allows selectively packaged release from cells. [46] Therefore, this helper-dependent system reduces toxicity but helps prolonged gene expression of up to 32 kb of foreign DNA in host cells. Nowadays, gutless adenovirus is administered in different organs, such as muscle, liver, and central nervous system. [45],[46],[47],[48],[49],[50],[51]
Hybrid adenoviral vectors
Hybrid adenoviral vectors are made of the high transduction efficiency of a gene-deleted adenoviral vector and the long-term genome-integrating potential of adeno-associated and retroviruses viruses. Such hybrid systems show stable transduction and limited integration sites. [52],[53] Among integrating vectors, those derived from retroviruses are most common. One of the family of Retroviridae are called spuma retroviruses or foamy viruses (FVs). FVs are a group of apparently nonpathogenic nonhuman retroviruses, which have been developed only recently. [54],[55] The potential advantages of FV vectors include a broad range of hosts, the largest packaging capacity of any retrovirus, and the ability to persist in quiescent cells. Because of these features, FVs have the unique potential to safely and efficiently deliver several genes into a number of different types of cells. [56],[57]
Herpes simplex virus
Herpes simplex virus (HSV) is one of the recent viruses candidate in gene delivery. HSV systems include the development of the so-called disabled infectious single copy (DISC) viruses, which comprise a glycoprotein H defective mutant HSV genome. When the defective HSV propagated in complementing cells' viral particles are generated, they can infect in subsequent cells permanently replicating their own genome but not producing more infectious particles. [58] Herpes vectors can deliver up to 150 kb transgenic DNA and because of its neuronotropic features, it has the greatest potential for gene delivery to nervous system, [59] tumors, and cancer cells. [60],[61],[62],[63],[64]
Lentiviruses
Lentiviruses are a subclass of retroviruses. They have recently been used as gene delivery vectors due to their ability to naturally integrate with nondividing cells, which is the unique feature of lentiviruses as compared with other retroviruses, which can infect only the dividing cells. Lentiviral vectors can deliver 8 kb of sequence. Because lentiviruses have strong tropism for neural stem cells, extensively used for ex vivo gene transfer in central nervous system with no significant immune responses and no unwanted side effects. Lentiviral vectors have the advantages of high-efficiency infection of dividing and nondividing cells, long-term stable expression of a transgene, low immunogenicity, and the ability to accommodate larger transgenes. [65],[66],[67]
There are numerous examples of effective long-term treatment of animal models of neurologic disorders, such as motor neuron diseases, Parkinson, Alzheimer, Huntington's disease, lysosomal storage diseases, and spinal injury. [68],[69],[70],[71],[72],[73]
Poxvirus vectors
Poxvirus vectors are members of the Poxviridae family that are widely used for high-level cytoplasmatic expression of transgenes. The high stable insertion capacity (more than 25 KB) of this virus is the most advantageous feature of it for gene delivery. The insertion of the transgene sequences is somewhat different from the other vector systems and utilizes homologous recombination or in vitro ligation for construction of recombinant vaccinia virus vectors. [74],[75],[76] Poxviruses have been used for cancer therapy in various studies, such as prostate cancer, colorectal cancer, breast cancer, and lung cancer. [77],[78] Recombinant vaccinia virus vectors were also used for expression of E6 and E7 0genes of human papilloma virus types 16 and 18 in cervical cancer patients to induce tumor regression. [79]
There are some problems in utilizing poxviruses for gene delivery because of their complex structure and biology, so further studies are required to improve their safety and to reduce the risk of cytopathic effects.
Epstein-Barr virus
Epstein-Barr virus as a herpes virus can be used for the expression of large DNA fragments in target cells. Because Epstein-Barr virus (EBV) establishes itself in the host nucleus in a latent state as extrachromosomal circular plasmid, this virus is suitable for long-term retention in the target cell. [80],[81],[82] Because of the natural B-cell tropism of the virus, EBV-derived vectors, such as B-cell lymphoma, have been tested for immune therapy of cancer. [83]
However, other types of viruses are under investigation to date and recently, many more different virus vector systems are being developed. These are derived from vaccinia virus, human cytomegalovirus, EBV, but as mentioned earlier, problems, such as their mutagen and carcinogen properties and long-term maintenance, are major limitations in utilizing the viral vectors in gene therapy.
| Nonviral Delivery Systems | | |
Nonviral systems comprise all the physical and chemical systems except viral systems and generally include either chemical methods, such as cationic liposomes and polymers, or physical methods, such as gene gun, electroporation, particle bombardment, ultrasound utilization, and magnetofection. Efficiency of this system is less than viral systems in gene transduction, but their cost-effectiveness, availability, and more importantly less induction of immune system and no limitation in size of transgenic DNA compared with viral system have made them more effective for gene delivery than nonviral delivery systems to date. [84],[85]
Physical methods of nonviral gene delivery
Physical methods applied for in vitro and in vivo gene delivery are based on making transient penetration in cell membrane by mechanical, electrical, ultrasonic, hydrodynamic, or laser-based energy so that DNA entrance into the targeted cells is facilitated.
Naked DNA
Naked DNA alone is able to transfer a gene (2-19 kb) into skin, thymus, cardiac muscle, and especially skeletal muscle and liver cells when directly injected, [86],[87] also it has been applied directly. [87] Long-term expression has been observed in skeletal muscle following injection for more than 19 months. Single injection yields transgenic expression in less than 1% of total myofibers of the muscle but multiple injection would improve it. Although naked DNA injection is a safe and simple method, its efficiency for gene delivery is low so it is only proper for some applications, such as DNA vaccination.
DNA particle bombardant by gene gun
DNA particle bombardant by gene gun is an ideal alternative technique to injection of naked DNA. Gold or tungsten spherical particles (1-3 μm diameter) are coated with plasmid DNA and then accelerated to high speed by pressurized gas to penetrate into target tissue cells. [88] Actually it is a modification of a technique called "biolistic," originally developed for plant transgenesis, but now used for in vitro and in vivo gene delivery into mammalian cells too, [89],[90] such as skin, mucosa, or surgically exposed tissue and especially for DNA-based immunization or vaccination. [91]
Electroporation
Electroporation is temporary destabilization of the cell membrane targeted tissue by insertion of a pair of electrodes into it so that DNA molecules in the surrounding media of the destabilized membrane would be able to penetrate into cytoplasm and nucleoplasm of the cell [92],[93] but unfortunately the trangene can integrate only to 0.01% of the treated cells. [94] Electroporation has been used in vivo for many types of tissues, such as skin, muscle, lung, [95],[96],[97] HPRT gene delivery, [98] and tumor treatment. [99] There are some problems in this method too that the more important are the difficulty in surgical procedure in the placement of electrodes into the internal tissues and that the high voltage applied to tissue might damage the organ and affect genomic DNA stability. [100]
Hydrodynamic
Hydrodynamic is a simple and highly efficient method for direct intracellular delivery of any water-soluble compounds and particles into internal organs. [101] The efficiency of this simple method in vivo is higher than any other nonviral system. This method has been successful for gene delivery into rodent liver and expression of hemophilia factors, [102] cytokines, [103] erythropoietin, [104] and hepatic growth factors, [105] in mouse and rat but it has been successful only in small animals and not in human.
Ultrasound
Ultrasound can make some nanomeric pores in membrane to facilitate intracellular delivery of DNA particles into cells of internal organs or tumors, so the size and concentration of plasmid DNA have great role in efficiency of the system. [106],[107] The most important limitation of the system is low efficiency of it, especially in vivo.
Magnetofection
Magnetofection is a simple and efficient transfection method that has the advantages of the nonviral biochemical (cationic lipids or polymers) and physical (electroporation, gene gun) transfection systems in one system while excluding their inconveniences, such as low efficiency and toxicity. In this method the magnetic fields are used to concentrate particles containing nucleic acid into the target cells. [108],[109] In this way, the magnetic force allows a very rapid concentration of the entire applied vector dose onto cells, so that 100% of the cells get in contact with a significant vector dose. Magnetofection has been adapted to all types of nucleic acids (DNA, siRNA, dsRNA, shRNA, mRNA, ODN,…), nonviral transfection systems (transfection reagents) and viruses. It has been successfully tested on a broad range of cell lines, hard-to-transfect and primary cells. [110],[111]
Chemical nonviral delivery systems
Chemical systems are more common than physical methods and generally are nanomeric complexes, which include compaction of negatively charged nucleic acid by polycationic nanomeric particles, belonging to cationic liposome/micelle or cationic polymers. The nanomeric complex between a cationic liposome or micelle and nucleic acids is called lipoplex; but polyplex is the nanomeric complex formed between a cationic polymer and nucleic acids. These nanomeric complexes are generally stable enough to produce their bound nucleic acids from degradation and are competent to enter cells usually by endocytosis. [112] Cationic nonviral delivery systems have several advantages compared to other nonviral systems and especially viral vectors, such as low toxicity and antigenicity because they are made of only biological lipids, long-term expression with less risk of insertional oncogenesis but still low efficiency is the disadvantage of this system as well. Generally cationic lipids are included in 6 subcategories:
(1) Monovalent cationic lipids
(2) Polyvalent cationic lipids
(3) Guanidine containing
(4) Cholesterol derivative compounds
(5) Cationic polymers: Poly(ethylenimine) (PEI)
Poly-l-lysine) (PLL)
Protamine
Other cationic polymers [113]
(6) Lipid-polymer hybrid
Mechanism of gene delivery by cationic particles
The mechanism of gene delivery by cationic systems includes 4 steps:
- Nonspecific interaction between cationic particles and cell surface
- Endocytosis into endocytosis vesicles (endosomes)
- Compaction and release of the DNA particle from endosomes
- Translocation of the DNA particle to nucleus by membrane receptors and transgenic expression of it. [114]
For targeting of cationic particles various cell-targeting legends are covalently attached to a lipid anchor (in lipoplexes) or a DNA-binding cationic polymer (in polyplexes), [115] including proteins, [116],[117],[118] antibodies, [119],[120] small chemical compounds, [121] carbohydrates, [122] peptide ligands, [123] and vitamins, [124] some of these ligands have enhanced the vector efficiency from 10- to 1000-folds. When lipoplex or polyplex particles made association with cell surface, they would enter the cell by endocytosis. It seems more of the lipid particles in early endosomes become trapped in lysosomes and degenerate by nucleases so the interaction of endosome with lysosome is a consensus and lipoplex or polyplex particles should be released before contraction of lysosome to endosome, so fugenic peptides can help it, these peptides originating from viruses can cut off the endosomal membrane to release the genomic DNA leading to increase of genetic translocation efficiency of the liposome. [125]
In this section we focus mainly on the 2 most common cationic particles: Cationic lipids and cationic polymers:
Cationic liposomes
Cationic liposomes are the more important current nonviral polycationic systems, which compact negatively charged nucleic acids lead to the formation of nanomeric complexes. Cationic liposomes have unique characteristics, such as capability to incorporate hydrophilic and hydrophobic drugs, low toxicity, no activation of immune system, and targeted delivery of bioactive compounds to the site of action. [126],[127],[128],[129] But the rapid degradation of liposomes due to the reticuloendothelial system and the inability to achieve sustained drug delivery over a prolonged period of time are 2 drawbacks of these delivery systems that have been overcome by modification of the surface of liposomes with hydrophilic polymers, such as polyethylene glycol (PEG) [128] and integration of the pre-encapsulated drug-loaded liposomes within depot polymer-based systems. [130]
All liposomes have 1 or 2 fatty acids and alkyl moieties that are 12-18 carbons in length, in addition to a positively charged polar head group hydrophobic groups, this hydrophobic structure causes the cationic lipids. Since the first monovalent cationic lipid, DOTAP, was synthesized by Felgner et al. in 1987, [131] hundreds of new cationic liposome/micelle systems have been reported for gene delivery in vitro or in vivo. The routine way to prepare a lipoplex is mixing the solution of plasmid DNA and liposome in a proper buffer. The gene delivery efficiency of liposomes is dependent on the size, structure, and even the amount of the liposome, the charge ratio between transgenic DNA and cationic liposome, presence of helper lipid, and the structure and proportion of it and cell type.
As mentioned earlier, cationic systems are mad of either a single synthetic cationic amphiphile (cytofectin), such as DOTAP, DOTMA, DOSPA, DOGS, or more commonly of a combination of a cationic amphiphile and a neutral lipid, such as DOPE and cholesterol, these neutral helper lipids unstabilize the endosomal membrane to facilitate lipid exchange and membrane fusion between lipoplexes and endosomal membrane leading to more gene expression. [132],[133] Cationic liposome-mediated delivery of DNA materials is optimal in vivo when the mol ratio of cationic liposome to nucleic acid in the lipoplex mixture is such that the positive/negative charge ratio is around 1 or greater [134],[135],[136] and in vitro the optimal ratio is closer to 1. [137],[138],[139],[140] However, multivalent lipids with long and unsaturated hydrocarbon chains are more efficient than monovalent cationic lipids with the same hydrophobic chains. [141]
Cationic liposomes are being used in gene delivery into lung, skeletal muscles, spleen, kidney, liver, testis, heart, and skin cells. [141],[142],[143],[144],[145],[146],[147],[148]
For gene transfer in vivo, many complexes (in equimolar ratios) are used that the more general ones are Chol/DOPE (1:1), DOTMA/DOPE (1:1), and DOTAP/DOPE (1:1).
Liposome-based technology has progressed from the first-generation conventional vesicles to stealth liposomes, targeted liposomes, and more recently stimuli-sensitive liposomes. [149],[150] These new generation of liposomes overcome most of the challenges encountered by conventional liposomes, such as the inability to escape from immune system, toxicity due to charged liposomes, and low half-life stability. [151],[152],[153]
Cationic polymers
Cationic polymers at first were introduced by Wu et al.1987 184 as PLL, the same year of synthesizing the first cationic lipids, and were further expanded by a second generation, PEI by Behr et al. in 1995. [154] To date a variety of linear or branched cationic polymers have been synthesized, including PLL-containing peptides, endosomolytic peptides (histidine-rich peptides), fusogenic peptides, nuclear localization peptides (mono partite NLS(Nuclear localization signal), bipartite NLS, nonclassical NLS), proteosomes. [155] However, PLL is still the most widely studied cationic polymer and has been used in a variety of polymerizations of lysine ranging from 19 to 1116 amino acid residues (3.97-233.2 kDa). While the molecular weight of the polymer increases, the net positive charge of it also increases and are therefore able to bind DNA tighter and form more stable complexes, totally. There is a relationship between the length of the polymer, gene delivery efficiency, and toxicity as the length of the polymer increases, so does its efficiency and its toxicity. [155],[156] However, the efficiency of PLL-mediated polyplexes are low when the PLL is used alone so some conjugation agents are used to facilitate cellular uptake in vitro (as EGF(fibroblast growth factor) or transferring) or endosomal escape in vivo (as fusogenic peptides or defective viruses). Also the attachment of PEG to the polymer can prevent plasma protein binding and increase circulation of half-life of the complex. [157],[158] Different homogenous PLL-conjugated peptides have been developed that have low toxicity, higher efficiency, and site-specific attachment of ligands used for cell targeting. [159],[160],[161],[162] The optimal peptide sequence contains 18 lysines followed by a tryptophan and alkylated cysteine (AlkCWK18). A variety of branched forms of cationic peptides with a lysine as branching point have been explored. [162] PEI is the most important cationic polymer next to PLL. PEI is one of the most positively charged dense polymers, synthesized in linear (LPEI) or branched (BPEI) form, which have high transfection activity in vitro and moderate activity in vivo but the linear forms have low toxicity and high efficiency than branched forms. [163] As PLL, conjugation of some agents, such as galactose, anti-CD3 antibodies and RGD motif-containing peptides can facilitate PEI polyplex cellular uptake. [164],[165],[166] Two advantages of PEI is that it forms toroidal polyplex particles, which are stable to aggregation in physiological buffer conditions, PEI also has a strong buffering capacity at almost any pH because of the great number of primary, secondary, and tertiary amino groups. [167] One disadvantage of PEI is its nonbiodegradable nature [168] and its serious toxicity in vivo (in contrast to cationic liposome/micelle). There are conflicting associations between the gene delivery efficiency and PEI toxicity, such as PLL, the most active PEI is 25 k for BPEI and 22 k for LPEI. [169] Unfortunately, due to this property there are some limitations in the application of PEI in nonviral vector in vivo delivery. More biodegradable cationic polymers, such as aminoesters have been explored that have less toxicity than PEI and PLL. [170] However, as mentioned earlier, there are a variety of new cationic polymer groups but each of them have some advantages and disadvantages. [155] The notable factors for in vivo application are toxicity and transfection efficiency.
Lipid-polymer systems
Lipid-polymer systems are 3-part systems in which DNA is first precondensed with polycations and then coated with either cationic liposomes, anionic liposomes, or amphiphilic polymers with or without helper lipids. [171],[172],[173],[174]
| Conclusion | | |
Although numerous viral and nonviral gene delivery systems have been developed in the last 3 decades, all of them have some disadvantages that have made some limitations in their clinical application and yet no delivery system has been designed that can be applied in gene therapy of all kinds of cell types in vitro and in vivo with no limitation and side effects; however, some delivery systems has been explored, which can be efficient for gene delivery to specific cells or tissues. So it seems that the process of developing successful delivery systems, especially nonviral systems, for use in in vivo is still in its adolescence and more efforts are needed. Totally, key steps effective in improving the currently available systems include the following: (1) improving extracellular targeting and delivery, (2) enhancing intracellular delivery and long-time expression, and (3) reducing toxicity and side effects on human body. However, clinical successes in 2009-2011 have bolstered new optimism in the promise of gene therapy. These include successful treatment of patients with the retinal disease Leber congenital amaurosis, [175],[176],[177],[178] X-linked SCID, [179] ADA-SCID, [180] adrenoleukodystrophy, [180] and Parkinson's disease. [181]
| References | | |
| 1. | Stone D. Novel viral vector systems for gene therapy. Viruses 2010;2:1002-7. 
[PUBMED] [FULLTEXT] |
| 2. | Katare DP, Aeri V. Progress in gene therapy: A review. I.J.T.P.R 2010;1:33.
|
| 3. | Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, et al. Gene transfer into humans immunotherapy of patients with advanced melanoma, using tumor- infiltrating lymphocytes modified by retroviral gene transduction. N Engl J Med 1990;323:570-8.
[PUBMED] [FULLTEXT] |
| 4. | Culver K, Cornetta K, Morgan R, Morecki S, Aebersold P, Kasid A, et al. Lymphocytes as cellular vehicles for gene therapy in mouse and man. Proc Natl Acad Sci USA 1991;88:3155-9.
[PUBMED] [FULLTEXT] |
| 5. | Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, et al. T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science 1995;270:475-80.
[PUBMED] [FULLTEXT] |
| 6. | Resnik B, Langer PJ. Human germline gene therapy reconsidered. Hum Gene Ther 2001;12:1449-58.
|
| 7. | McDonough PG. The ethics of somatic and germline gene therapy. Ann N Y Acad Sci 1997;816:378-82.
[PUBMED] [FULLTEXT] |
| 8. | Resnik DB, Langer PJ. Human germline gene therapy reconsidered. Hum Gene Ther 2001;12:1449-58.
[PUBMED] [FULLTEXT] |
| 9. | Wolf E, Schernthaner W, Zakhartchenko V, Prelle K, Stojkovic M, Brem G. Transgenic technology in farm animals-progress and perspectives. Exp Physiol 2000;85:615-25.
[PUBMED] [FULLTEXT] |
| 10. | Johnson-Saliba M, Jans DA. Gene therapy: Optimising DNA delivery to the nucleus. Curr Drug Targets 2001;2:371-99.
[PUBMED] [FULLTEXT] |
| 11. | Jaenisch R. Transgenic animals. Science 1988;240:1468-74.
[PUBMED] [FULLTEXT] |
| 12. | Smith KR. Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline. Int J Med Sci 2004;1:76-91.
[PUBMED] [FULLTEXT] |
| 13. | Hirabayashi M, Takahashi R, Ito K, Kashiwazaki N, Hirao M, Hirasawa K, et al. A comparative study on the integration of exogenous DNA into mouse, rat, rabbit, and pig genomes. Exp Anim 2001;50:125-31.
[PUBMED] [FULLTEXT] |
| 14. | Torres M. The use of embryonic stem cells for the genetic manipulation of the mous. Curr Topics Dev Biol 1998;36:99-114.
[PUBMED] |
| 15. | Gordon JW, Scangos GA, Plotkin DJ, Barbosa JA, Ruddle FH. Genetic transformation of mouse embryos by micro-injection of purified DNA. Proc Natl Acad Sci USA 1980;77:7380-4.
[PUBMED] [FULLTEXT] |
| 16. | John PL, Kevin C, Joaquin G. Sperm and testis mediated DNA transfer as a means of gene therapy. Syst Biol Reprod Med 2011;57:35-42.
|
| 17. | Kevin S, Corrado S. Sperm-mediated gene transfer: Applications and implications. BioEssays 2005;27:551-62.
|
| 18. | Herrero MJ, Sabater L, Guenechea G, Sendra L, Montilla AI, Abargues R, et al. DNA delivery to 'ex vivo' human liver segments. Gene Ther 2011 in press.
|
| 19. | Suhonen J, Ray J, Blömer U, Gage FH, Kaspar B. Ex vivo and in vivo gene delivery to the brain. Curr Protoc Hum Genet 2006;13:Unit 13.3.
|
| 20. | Hu WW, Wang Z, Hollister SJ, Krebsbach PH. Localized viral vector delivery to enhance in situ regenerative gene therapy. Gene Ther 2007;14:891-901.
[PUBMED] [FULLTEXT] |
| 21. | Takefumi S, Akira I, Shin E, Shiro B. In Situ Gene Therapy for Prostate Cancer. Curr Gene Ther 2005;5:111-9.
|
| 22. | Davis PB, Cooper MJ. Vectors for airway gene delivery. AAPS J 2007;9:2.
[PUBMED] [FULLTEXT] |
| 23. | Huang Y, Liu X, Dong L, Liu Z, He X, Liu W. Development of Viral Vectors for Gene Therapy for Chronic Pain. Pain Res Treat 2011;2011:968218.
|
| 24. | Gardlik R, Palffy R, Hodosy J, Lukacs J, Turna J, Celec P. Vectors and delivery systems in gene therapy. Med Sci Monit 2005;11:110-21.
|
| 25. | Katare DP, Aeri V. Progress in gene therapy: A Review. I.J.T.P.R. 2010;1:33-41.
|
| 26. | Wickham TJ. Ligand-directed targeting of genes to the site of disease. Nat Med 2003;9:135-9.
[PUBMED] [FULLTEXT] |
| 27. | Anson DS. The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery. Genet Vaccines Ther 2004;2:9.
[PUBMED] [FULLTEXT] |
| 28. | Frederic D. Bushman. Retroviral integration and human gene therapy. J Clin Invest 2007;117:2083-6.
|
| 29. | Laufs S, Gentner B, Nagy KZ, Jauch A, Benner A, Naundrof S, et al. Retroviral vector integration occurs in preferred genomic targets of human bone marrow-repopulating cells. Blood 2003;101:2191-8.
|
| 30. | Hacein-Bey-Abina S, Le Deist F, Carlier F, Bouneaud C, Hue C, De Villartay JP, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-93.
[PUBMED] [FULLTEXT] |
| 31. | Fischer A, Hacein-Bey-Aina S, Lagresle C, Garrigue A, Cavazanna-Calvo M. Gene therapy of severe combined immunodeficiency disease: Proof of principle of efficiency and safety issues. Gene therapy, primary immunodeficiencies, retrovirus, lentivirus, genome. Bull Acad Natl Med 2005;189:779-85.
|
| 32. | Buckly RH. Gene therapy for SCID: A complication after remarkable progress. Lancet 2003;360:1185-6.
|
| 33. | Fox JL. US authorities uphold suspension of SCID gene therapy. Nat Biotechnol 2003;21:217.
|
| 34. | Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-XI. Science 2003;302:415-9.
[PUBMED] [FULLTEXT] |
| 35. | Thrasher AJ, Gaspar HB, Baum C, Modlich U, Schambach A, Candotti F, et al. Gene therapy: X-SCID transgene leukaemologenicity. Nature 2006;443:E5-7.
[PUBMED] [FULLTEXT] |
| 36. | Vorburger SA, Hunt KK. Adenoviral Gene Therapy. Oncologist 2002;7:46-59.
[PUBMED] [FULLTEXT] |
| 37. | Bett AJ, Prevec L, Graham FL. Packaging capacity and stability of human adenovirus type 5 vectors. J Virol 1993;67:5911-21.
[PUBMED] [FULLTEXT] |
| 38. | Reid T, Warren R, Kirn D. Intravascular adenoviral agents in cancer patients: Lessons from clinical trials. Cancer Gene Ther 2002;9:979-86.
[PUBMED] [FULLTEXT] |
| 39. | Raper SE, Chirmule N, Lee FS, Wivel NA, Bagg A, Gao GP, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80:148-58.
[PUBMED] [FULLTEXT] |
| 40. | Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999;286:2244-5.
|
| 41. | Teramato S, Ishii T, Matsuse T. Crisis of adenoviruses in human gene therapy. Lancet 2000;355:1911-2.
[PUBMED] [FULLTEXT] |
| 42. | Lai CM, Lai YK, Rakoczy PE. Adenovirus and adeno-associated virus vectors. DNA Cell Biol 2002;21:895-913.
[PUBMED] [FULLTEXT] |
| 43. | Flotte T, Carter B, Conrad C, Guggino W, Reynolds T, Rosenstein B, et al. A phase study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease. Hum Gene Ther 1996;7:1145-59.
[PUBMED] [FULLTEXT] |
| 44. | Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000;24:257-61.
[PUBMED] [FULLTEXT] |
| 45. | Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration Molecular therapy. J Am Soc Gene Ther 2010;18:643-50.
[PUBMED] [FULLTEXT] |
| 46. | Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, et al. Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med 2009;361:725-7.
[PUBMED] [FULLTEXT] |
| 47. | Crane B, Luo X, Demaster A, Williams KD, Kozink DM, Zhang P, et al. Rescue administration of a helper dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia. Gene Ther 2011; in press.
|
| 48. | Alba R, Bosch A, Chillon M. Gutless adenovirus: Last-generation adenovirus for gene therapy. Gene Ther 2005;12:18-S27.
[PUBMED] [FULLTEXT] |
| 49. | Croyle MA, Le HT, Linse KD, Cerullo V, Toietta G, Beaudet A, et al. PEGylated helper-dependent adenoviral vectors: Highly efficient vectors with an enhanced safety profile. Gene Ther 2005;12:579-87.
[PUBMED] [FULLTEXT] |
| 50. | Amalfitano A, Hauser MA, Hu H, Serra D, Begy CR, Chamberlain JS. Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted. J Virol 1998;72:926-33.
[PUBMED] [FULLTEXT] |
| 51. | Morral N, O'Neal W, Rice K, Leland M, Kaplan J, Piedra PA, et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci USA 1999;96:12816-21.
[PUBMED] [FULLTEXT] |
| 52. | Balagué C, Zhou J, Dai Y, Alemany R, Josephs SF, Andreason G, et al. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 2000;95:820-8.
|
| 53. | Morral N, Parks RJ, Zhou H, Langston C, Schiedner G, Quinones J, et al. High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity. Hum Gene Ther 1998;9:2709-16.
|
| 54. | Ehrhardt A, Yant SR, Giering JC, Xu H, Engler JA, Kay MA. Somatic Integration From an Adenoviral Hybrid Vector into a Hot Spot in Mouse Liver Results in Persistent Transgene Expression Levels In Vivo. Mol Ther 2007;15:146-56.
|
| 55. | Shuji K, Kazunori H, Atsuko T, Donna JP, Philip N, Haruki O, et al. Adenovirus-retrovirus hybrid vectors achieve highly enhanced tumor transduction and antitumor efficacy in vivo. Mol Ther 2011;19:76-82.
|
| 56. | Liu W, Liu Z, Cao X, Cao Z, Xue L, Zhu F, et al. Recombinant human foamy virus, a novel vector for neurological disorders gene therapy, drives production of GAD in cultured astrocytes. Mol Ther 2007;15:1834-41.
|
| 57. | Mergia A, Heinkelein M. Foamy virus vectors. Curr Topics Microbiol Immunol 2003;277:131-59.
|
| 58. | Trobridge GD. Foamy virus vectors for gene transfer. Exp Opin Biol Ther 2009;9:1427-36.
|
| 59. | Liu W, Liu Z, Liu L, Xiao Z, Cao X, Cao Z, et al. A novel human foamy virus mediated gene transfer of GAD67 reduces neuropathic pain following spinal cord injury. Neurosci Lett 2008;432:13-8.
|
| 60. | Burtton EA, Wechuck JB, Wendell SK, Goins WF, Fink DJ, Glorioso JC. Multiple applications for replication-defective Herpes simplex virus vectors. Stem Cell 2001;19:358-77.
|
| 61. | Berto E, Bozac A, Marconi P. Development and application of replication incompetent HSV-1-based vectors. Gene Ther 2005;12:98-102.
|
| 62. | Goins WF, Goss JR, Chancellor MB, de Groat WC, Glorioso JC, Yoshimura N. Herpes simplex virus vectormediated gene delivery for the treatment of lower urinary tract pain. Gene Ther 2009;16:558-69.
|
| 63. | Wolfe D, Mata M, Fink DJ. A human trial of HSVmediated gene transfer for the treatment of chronic pain. Gene Ther 2009;16:455-60.
|
| 64. | Goss JR, Harley CF, Mata M, O'Malley ME, Goins WF, Hu X, et al. Herpes vectormediated expression of proenkephalin reduces bone cancer pain. Ann Neurol 2002;52:662-5.
|
| 65. | Goss JR, Mata M, Goins WF, Wu HH, Glorioso JC, Fink DJ. Antinociceptive effect of a genomic herpes simplex virus-based vector expressing human proenkephalin in rat dorsal root ganglion. Gene Ther 2001;8:551-6.
|
| 66. | Lachmann RH, Efstathiou S. The use of Herpes simolex virus-based vectors for gene delivery to the nervous system. Mol Med Today 1997;3:404-11.
|
| 67. | Federici T, Kutner R, Zhang XY, Kuroda H, Tordo N, Boulis NM, et al. Comparative analysis of HIV-1-based lentiviral vectors bearing lyssavirus glycoproteins for neuronal gene transfer. Genet Vaccines Ther 2009;7:1-9.
|
| 68. | Cockrell AS, Kafri T. Gene delivery by lentivirus vectors. Mol Biotechnol 2007;36:184-204.
|
| 69. | Kafri T. Gene delivery by lentivirus vectors an overview. Methods Mol Biol 2004;246:367-90.
|
| 70. | Balaggan KS, Ali RR. Ocular gene delivery using lentiviral vectors. Gene Ther 2012;19:145-53.
|
| 71. | Wong LF, Goodhead L, Prat C, Mitrophanous KA, Kingsman SM, Mazarakis ND. Lentivirus-Mediated Gene Transfer to the Central Nervous System: Therapeutic and Research Applications. Human Gen Ther 2006;17:1-9.
|
| 72. | Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, et al. Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J Neurosci 2002;22:10302-12.
|
| 73. | Betchen SA, Kaplitt M. Future and current surgical therapies in Parkinson's disease. Curr Opin Neurol 2003;16:487-93.
|
| 74. | Blesch A, Ynski MH. Gene therapy and cell transplantation for Alzheimer's disease and spinal cord injury. Yonsei Med J 2004;45:28-31.
|
| 75. | Singer O, Marr RA, Rockenstein E, Crews L, Coufal NG, Gage FH, et al. Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat Neurosci 2005;8:1343-9.
|
| 76. | Moroziewicz D, Kaufman HL. Gene therapy with poxvirus vectors. Curr Opin Mol Ther 2005;7:317-25.
|
| 77. | Gómez CE, Nájera JL, Krupa M, Esteban M. The poxvirus vectors MVA and NYVAC as gene delivery systems for vaccination against infectious diseases and cancer. Curr Gene Ther 2008;8:97-120.
|
| 78. | Pastoret PP, Vanderplasschen A. Poxviruses as vaccine vectors. Comp Immunol Microbiol Infect Dis 2003;26:343-55.
|
| 79. | Moss B. Genetically engineered poxviruses for recombinant gene expression, vaccination and safety. Proc Natl Acad Sci U S A 1996;93:11341-8.
|
| 80. | McAneny D, Ryan CA, Beazley RM, Kaufman HL. Results of a phase I trial of a recombinant vaccinia virus that expresses carcinoembryonic antigen in patients with advanced colorectal cancer. Ann Surg Oncol 1996;3:395-500.
|
| 81. | Borysiewicz LK, Fiander A, Nimako M, Man S, Wilkinson GW, Westmoreland D, et al. A recombinant vaccinia virus encoding human papillomavirus types 16 and 18, E6 and E7 proteins as immunotherapy for cervical cancer. Lancet 1996;347:1523-7.
|
| 82. | Mecsas J, Sugden B. Replication of plasmids derived from bovine papilloma virus type 1 and Epstein-Barr virus in cell in culture. Annu Rev Cell Biol 1987;3:87-108.
|
| 83. | Kishida T, Shin-Ya M, Imanishi J, Mazda O. Dept. of Microbiol., Kyoto Prefectural Univ. of Medicine Application of EBV-based artificial chromosome to genetic engineering of mammalian cells and tissues. Micro Nano Mechatron Hum Sci 2005;7-9:133-8.
|
| 84. | Hirai H, Satoh E, Osawa M, Inaba T, Shimazaki C, Kinoshita S, et al. Use of EBV-based vector/ HVJ-liposome complex vector for targeted gene therapy of EBV-associated neoplasms. Biochem Biophys Res Commun 1997;241:112-8.
|
| 85. | Robertson ES, Ooka T, Kieff ED. Epstein-Barr virus vectors for gene delivery to B lymphocytes. Proc Natl Acad Sci U S A. 1996;93:11334-40.
|
| 86. | Audouny SA, Deleij LF, Hoekstra D, Molema G. In vivo characteristics of cationic liposomes as delivery vectors for gene therapy. Pharm Res 2002;19:1599-6005.
|
| 87. | Varga CM, Hong K, Lanf Furburger DA. Quantitative analysis of synthesis gene delivery vector design properties. Mol Ther 2001;4:438-46.
|
| 88. | Wolff JA, Ludtke JJ, Acsadi G, Williams P, Jani A. Long term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum Mol Genet 1992;1:363-9.
|
| 89. | Knapp JE, Liu D. Hydrodynamic delivery of DNA. Methods Mol Biol 2004;245:245-50.
|
| 90. | Herweijer H, Wolff JA. Progress and prospects: Naked DNA gene transfer and therapy. Gen Ther 2003;10:453-8.
|
| 91. | Yang NS, Burkhorder J, Roberts B, Martinell B, McCabe D. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci USA 1990;87:9568-72.
|
| 92. | Klein TM, Arentzen R, Lewis PA, Fitzpatrick McElligoutt S. Transformation of microbes, plants and animals by particle bombardment. Biotechnology 1992;10:286-91.
|
| 93. | Cheng L, Ziegelhoffer PR, Yang NS. In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci USA 1993;90:4455-9.
|
| 94. | Mahvi DM, Sheehy MJ, Yang NS. DNA cancer vaccines: A gene gun approach. Immunol Cell Biol 1997;75:456-60.
|
| 95. | Heller LC, Ugen K, Heller R. Electroporation for targeted gene transfer. Expert Opin Drug Deliv 2005;2:255-68.
|
| 96. | Lurquin PF. Gene transfer by electroporation. Mol Biotechnol 1997;7:5-35.
|
| 97. | Potter H, Cooke SW. Gene transfer into adherent cells growing on microbeads. In: Change DC, editor. Guide to electroporation and electrofusion. San Diego, CA, USA: Academic Press; 1992. p. 201-8.
|
| 98. | Neumann E, Schaefer-Ridder M, Wang Y, Hofschneider PH. Gene transferin to mouse lyoma cells by electroporation in high electric fields. EMBOJ 1982;1:841-5.
|
| 99. | Dean DA, Machado Aranda D, Blair Parks K, Yeldandi AV, Young JL. Electroporation as a method for high level nonviral gene transfer to the lung. Gene Ther 2003;10:1608-15.
|
| 100. | Mc Mahon JM, Wells DJ. Electroporation for gene transfer to skeletal muscles: Current status. Biol Drugs 2004;18:155-65.
|
| 101. | Hatada S, Nikkuni K, Bentley SA, Kirby S, Smithies O. Gene correction in hematopoietic progenitor cells by homologous recombination. Proc Natl Acad Sci USA 2000;97:13807-11.
|
| 102. | Hofmann GA, Dev SB, Nanda GS, Rabusssay D. Electroporation therapy of solid tumors. Crit Rev Ther Drug Carrier Syst 1999;16:523-69.
|
| 103. | Gissel H, Clausen T. Excitation -induced Ca influx and skeletal muscle cell damage. Acta Physiol Scand 2001;171:327-34.
|
| 104. | Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999;10:1258-66.
|
| 105. | Miao CH, Ye X, Thompson AR. High level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Hum Gen Ther 2003;14:1297-305.
|
| 106. | Jiang J, Yamato E, Miyazaki J. Intravenous delivery of naked plasmid DNA for in vivo cytokine expression. Biochem Biophys Res Commun 2001;289:1088-92.
|
| 107. | Maruyama H, Higuchi N, Kameda S, Miyazaki J, Gejyo F. Rat liver targeted naked plasmid DNA transfer by tail vein injection. Mol Biotechnol 2004;26:165-72.
|
| 108. | Yang J, Chen S, Huang L, Michalopoulos GK, Liu Y. Sustained expression of naked plasmid DNA encoding hepatocyte growth factor in mice promotes liver and overall body growth. Hepatology 2001;33:848-59.
|
| 109. | Kim HJ, Greeenleaf JF, Kinnick RR, Bronk JT, Bolander ME. Ultrasound-mediated transfection of mammalian cells. Hum Gene Ther 1996;7:1339-46.
|
| 110. | Liang HD, Lu QL, Xue SA, Halliwell M, Kodama T, Cosgrove DO, et al. Optimisation of ultrasound mediated gene transfer (sonoporation) in skeletal muscle cells. Ultrasound Med Biol 2004;30:1523-9.
|
| 111. | Plank C, Schillinger U, Scherer F, Bergemann C, Rémy JS, Krötz F, et al. The magnetofection method: Using magnetic force to enhance gene delivery. Biol Chem 2003;384:737-47.
|
| 112. | Scherer F, Anton M, Schillinger U, Henke J, Bergemann C, Krüger A, et al. Magnetofection: Enhancing and targeting gene delivery by magnetic force in vitro and in vivo. Gene Ther 2002;9:102-9.
|
| 113. | Plank C, Anton M, Rudolph C, Rosenecker J, Krötz F. Enhancing and targeting nucleic acid delivery by magnetic force. Exp Opin Biol Ther 2003;3:745-58.
|
| 114. | Mair L, Ford K, Alam MR, Kole R, Fisher M, Superfine R. et al. Size-Uniform 200 nm Particles: Fabrication and Application to Magnetofection. J Biomed Nanotechnol 2009;5:182-91.
|
| 115. | Liu D, Ren T, Gao X. Cationic transfection lipids. Curr Med Chem 2003;10:1735-7.
|
| 116. | Zhang S, Xu Y, Wang B, Qiao W, Liu D, Li Z. Cationic compounds used in lipoplexes and polyplexes for gene delivery. J Controlled Release 2004;100:165-80.
|
| 117. | Khalil IA, Kogure K, Akita H, Harashima H. Uptake pathways and subsequent intracellular trafficking in nonviral gene delivery. Pharmacol Rev 2006;58:32-45.
|
| 118. | Boeckle S, Wagner E. Optimizing targeted gene delivery: Chemical Modification of viral vectors and synthesis of artificial virus vector systems. AAPS J 2006;8:Article 83.
|
| 119. | Kim TG, Kang SY, Kang JH, Cho MY, Kim JI, Kim SH, et al. Gene transfer into human hepatoma cells by receptor-associated protein/polylysine conjugates. Bioconjug Chem 2004;15:326-32.
|
| 120. | Wolschek MF, Thallinger C, Kursa M, Rössler V, Allen M, Lichtenberger C, et al. Specific systemic nonviral gene delivery to human hepatocellular carcinoma xenografts in SCID mice. Hepatology 2002;36:1106-14.
|
| 121. | Xu L, Pirollo KF, Tang WH, Rait A, Chang EH. Transferrin-liposome-mediated systemic p53 gene therapy in combination with radiation results in regression of human head and neck cancer xenografts. Hum Gene Ther 1999;10:2941-52.
|
| 122. | Chiu SJ, Ueno NT, Lee RJ. Tumor-targeted gene delivery via anti-HER2 antibody (trastuzumab, Herceptin) conjugated polyethylenimine. J Control Release 2004;97:357-69.
|
| 123. | Zhang Y, Zhang YF, Bryant J, Charles A, Boado RJ, Pardridge WM. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin Cancer Res 2004;10:3667-77.
|
| 124. | Hood JD, Bednarski M, Frausto R, Guccione S, Reisfeld RA, Xiang R, et al. Tumor regression by targeted gene delivery to the neovasculature. Science 2002;296:2404-7.
|
| 125. | Plank C, Zatloukal K, Cotton M, Mechtler K, Wagner E. Gene transfer into hepatocytes using asialoglycoprotein receptor-mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand1. Bioconjug Chem 1992;3:533-9.
|
| 126. | Ziandy AG, Ferkol T, Dawson DV, Perlmutter DH, Davis PB. Chain length of the polylysine in receptor-targeted gene transfer complexes affects duration of reporter gene expression both in vitro and in vivo. 1999;274:4908-16.
|
| 127. | Hofland HE, Masson C, Iginla S, Osetinsky I, Reddy JA, Leamon CP, et al. Folate-targeted gene transfer in vivo. Mol Ther 2002;5:739-44.
|
| 128. | Son KK, Tkaeh D, Hall KJ. Efficient in vivo gene delivery by the negatively charged complexes of cationic liposome and plasmid. DNA Biochem Biophys Acta 2000;1468:6-10.
|
| 129. | Mastrobattista E, Koning GA, van Bloois L, Filipe AC, Jiskoot W, Storm G. Functional characterization of an endosome-disruptive peptide and its application in cytosolic delivery of immunoliposome-entrapped proteins. J Biol Chem 2002;277:27135-43.
|
| 130. | Schnyder A, Huwyler J. Drug transport to brain with targeted liposomes. NeuroRx 2005;2:99-107.
|
| 131. | Immordino ML, Dosio F, Cattel L. Stealth liposomes: Review of the basic science, rationale, and clinical applications, existing and potential. Int J Nanomed 2006;1:297-315.
|
| 132. | Chen C, Han D, Cai C, Tang X. An overview of liposome lyophilization and its future potential. J Control Release 2010;142:299-311.
|
| 133. | Stenekes RJ, Loebis AE, Fernandes CM, Crommelin DJ, Hennink WE. Controlled release of liposomes from biodegradable dextran microspheres: A novel delivery concept. Pharm Res 2000;17:690-5.
|
| 134. | Felgner PL, Gadek TR, Holm M, Roman R, Chan HW, Wenz M, et al. Lipofection: A highly efficient, lipid-mediated DNA-transfection procedure. Proc Natl Acad Sci USA 1987;84:7413-7.
|
| 135. | Cullis PR, Hope MJ, Tilcock CP. Lipid polymorphism and the roles of lipids in membranes. Chem Phys Lipds 1986;40:127-44.
|
| 136. | Wrobel I, Collins D. Fusion of cationic liposomes with mammalian cells occurs after endocytosis. Biochem Biophys Acta 1995;1235:296-304.
|
| 137. | Schwartz B, Benoist C, Abdallah B, Scherman D, Behr JP, Demeneix BA. Liposopermine-based gene transfer into the newborn mous brain is optimized by a low lipospermine DNA charge ratio. Hum Gene Ther 1995;6:1515-24.
|
| 138. | Liu F, Qi H, Huang L. Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther 1997;4:517-23.
|
| 139. | Yang JP, Huang L. Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther 1995;92:1744-1748.
|
| 140. | Felgner JH, Kumar R, Sridhar CN, Wheeler CJ, Tsai YJ, Border R, et al. Enhanced gene delivery and mechanism studies with a novol series of cationic lipid formulations. J Biol Chem 1994;269:2550-61.
|
| 141. | Alton EW, Middleton PG, Caplen NJ, Smith SN, Steel DM, Munkonge FM, et al. Non-invasive liposome mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nat Gent 1993;5:135-42.
|
| 142. | McQuillin A, Murray KD, Etheridge CJ, Stewart L, Cooper RG, Brett PM, et al. Optimization of liposome mediated transfection of a neuronal cell line. 1997;8:135-42.
|
| 143. | Fife K, Bower M, Cooper RG. Endothelial cell transfection with cationic liposomes and herpes simolex-thymidine kinase mediated killing. Gen Ther 1998;5:614-20.
|
| 144. | Birchall JC, Kellawy IW, Mills SN. Physicochemical characterization and transfection efficiency of lipid-based gene delivery complexes. Int J Pharm 1999;183:195-207.
|
| 145. | Stribling R, Brunette E, Liggitt D, Gaensler K, Debs R. Aerosol gene delivery in vivo. Proc Natl Acad Sci USA 1992;89:11277-81.
|
| 146. | Caplen NJ, Alton EW, Middleton PG, Dorin JR, Stevenson BJ, Gao X, et al. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nat Med 1995;1:39-46.
|
| 147. | Alton EW, Stern M, Farley R, Jaffe A, Chadwick SL, Phillips J, et al. Cationic lipid mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: A double blind placebo-controlled trial. Lancet 1999;353:947-54.
|
| 148. | Zhu N, Liggitt D, Liu Y, Debs R. Systemic gene expression after intravenous DNA delivery into adult mice. Science 1993;261:209-11.
|
| 149. | Rogy MA, Auffenberg T, Espat NJ, Philip R, Remick D, Wollenberg GK, et al. Human tumor necrosis factor receptor(p55) and interleukin 10 gene transfer in the mouse reduces mortality to the lethal endotoxemia and also attenuates local inflammatory responses. J Exp Med 1995;181:2289-93.
|
| 150. | Murray KD, McQuillin A, Stewart L, Etheridge CJ, Cooper RG, Miller AD, et al. Cationic liposome mediated DNA transfection in organotype explant cultures of the ventral mesencephalon. Gene Ther 1999;6:190-7.
|
| 151. | Thierry AR, Lunardiskander Y, Bryant JL, Rabinovich P, Gallo RC, Mahan LC. Systemic gene therapy: Biodistribution and long term expression of a transgene in mice. Proc Natl Acad Sci USA 1995;92:9742-6.
|
| 152. | Immordino ML, Dosio F, Cattel L. Stealth liposomes: Review of the basic science, rationale, and clinical applications, existing and potential. Int J Nanomed 2006;1:297-315.
|
| 153. | Bharali DJ, Khalil M, Gurbuz M, Simone TM, Mousa SA. Nanoparticles and cancer therapy: A concise review with emphasis on dendrimers. International. J Nanomed 2009;4:1-7.
|
| 154. | Kim ES, Lu C, Khuri FR, Tonda M, Glisson BS, Liu D, et al. A phase II study of STEALTH cisplatin (SPI-77) in patients with advanced nonsmall cell lung cancer. Lung Cancer 2001;34:427-32.
|
| 155. | Goyal P, Goyal K, Kumar SG, Singh A, Katare OP, Mishra DN. Liposomal drug delivery systems-clinical applications. Acta Pharm 2005;55:1-25.
|
| 156. | Pradhan P, Giri J, Rieken F, Koch C, Mykhaylyk O, Döblinger M, et al. Targeted temperature sensitive magnetic liposomes for thermo-chemotherapy. J Control Release 2010;1:108-21.
|
| 157. | Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B, et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: Polyethylenamine. Proc Natl Acad Sci USA 1995;92:7297-301.
|
| 158. | Martin ME, Rice KG. Peptide-guided Gene Delivery. AAPS J 2007;9:Article 3.
|
| 159. | Wolfert MA, Saymour LW. Atomic force microscopic analysis of the influence of the molecular weight of poly(L)lysine on the size of polyelectrolyte complexes formed with DNA. Gen Ther 1996;3:269-73.
|
| 160. | El-Aneed A. An overview of current delivery systems in cancer gene therapy. J Control Release 2004;94:1-14.
|
| 161. | Tang MX, Szoka FC. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther 1997;4:823-32.
|
| 162. | Wadhwa MS, Collard WT, Adami RC, McKenzie DL, Rice KG. Peptide-mediated gene delivery: Influence of peptide structure on gene expression. Bioconjug Chem 1997;8:81-8.
|
| 163. | McKenzie DL, Collard WT, Rice KG. Comparative gene transfer efficiency of low molecular weight polylysine DNA-condensing peptides. J Pept Res 1999;54:311-8.
|
| 164. | Adami RC, Collard WT, Gupta SA, Kwok KY, Bonadio J, Rice KG. Stability of peptide-condensed Plasmid DNA formulations. J Pharm Sci 1998;87:678-83.
|
| 165. | Plank C, Tang MX, Wolfe AR, Szoka FC. Branched cationic peptides for gene delivery: Role of type and number of cationic residues in formation and in vitro activity of DNA polyplexes. Hum Gene Ther 1999;10:319-32 .
|
| 166. | Wightman L, Kircheis R, Rössler V, Carotta S, Ruzicka R, Kursa M, et al. Different behavior of the branched and linear polyethylenamine for gene delivery in vitro and in vivo. J Gene Med 2000;3:362-72.
|
| 167. | Zanta MA, Boussif O, Adib A, Behr JP. In vitro gene delivery to hepatocytes with galactosylenimine. Bioconjugate Chem 1997;8:839-44.
|
| 168. | Kircheis R, Kichler A, Wallner G, Kursa M, Ogris M, Felzmann T, et al. Coupling of cell binding ligands to polyethylenimine for targeted gene delivery. Gene Ther 1997;4:409-18.
|
| 169. | Erbacher P, Remy JS, Behr JP. Gene transfer with synthetic virus like particles via the integrin mediated endocytosis pathway. Gene Ther 1999;6:138-45.
|
| 170. | Tang MX, Szoka FC. The influence of polymer structure on the interaction of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther 1997;4:823-32.
|
| 171. | Fischer D, Li Y, Ahlemeyer B, Krieglstein J, Kissel T. In vitro cytotoxicity testing of polycations: Influence of polymer structure on cell viability and hemolysis. Biomaterials 2003;24:1121-31.
|
| 172. | Fischer D, Bieber T, Li Y, Elasser HP, Kissel T. A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenamine: Effect of molecular weight on transfection efficiency and cytotoxicity. Pharm Res 1999;16:1273-9.
|
| 173. | Lim YB, Han SO, Kong HU. Biodegradable polyester, poly[alpha-(4-aminobutyl)-L-glycolic acid], as anon-toxic gene carrier. Pharm Res 2000;7:811-6.
|
| 174. | Gao X, Huang L. Potentiation of cationic liposome-mediated gene delivery by polycationics. Biochemistry 1996;35:1027-36.
|
| 175. | Lee RJ, Huang L. Folate-targeted, anionic liposome-entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J Biol Chem 1996;271:8481-7.
|
| 176. | Lee LK, Williams CL, Devore D, Roth CM. Poly (eropylacrylicacid) enhances cationic lipid-mediated delivery of antisense oligonucleotides. Biomacromolecules 2006;7:1502-8.
|
| 177. | Maguire AM, Simonelli F, Pierce EA, Pugh EN, Mingozzi F, Bennicelli J, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-8.
|
| 178. | Simonelli F, Maguire AM, Testa F, Pierce EA, Mingozzi F, Bennicelli JL, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration Molecular therapy. J Am Soc Gene Ther 2010;18:643-50.
|
| 179. | Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, et al. Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med 2009;361:725-7.
|
| 180. | Cartier N, Aubourg P. Hematopoietic Stem Cell Transplantation and Hematopoietic Stem Cell Gene Therapy in X-Linked Adrenoleukodystrophy. Brain Pathol 2010;20:857-862.
|
| 181. | LeWitt PA, Rezai AR, Leehey MA, Ojemann SG, Flaherty AW, Eskandar EN, et al. AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind, sham-surgery controlled, randomised trial. Lancet Neurol 2011;10:309-19.
|
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|
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|
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|
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|
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|
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|
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|
| Murtaza S. Nagree,Simone Scalia,William M. McKillop,Jeffrey A. Medin | | Expert Opinion on Biological Therapy. 2019; : 1 | | [Pubmed] | [DOI] | | | 46 |
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|
| Genghao Chen,Dhruva Katrekar,Prashant Mali | | Biochemistry. 2019; 58(15): 1947 | | [Pubmed] | [DOI] | | | 47 |
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|
| Jiang-Hu Huang,Yang Xu,Xiao-Ming Yin,Fei-Yue Lin | | Neuroscience. 2019; | | [Pubmed] | [DOI] | | | 48 |
Anti-ageing gene therapy: Not so far away? |
|
| Alexander Vaiserman,Elena De Falco,Alexander Koliada,Olga Maslova,Carmela Rita Balistreri | | Ageing Research Reviews. 2019; 56: 100977 | | [Pubmed] | [DOI] | | | 49 |
Advances in Targeted Gene Delivery |
|
| Anjuman A. Begum,Istvan Toth,Waleed M. Hussein,Peter M. Moyle | | Current Drug Delivery. 2019; 16(7): 588 | | [Pubmed] | [DOI] | | | 50 |
Synthetic promoter for efficient and muscle-specific expression of exogenous genes |
|
| Yili Liu,Yutong He,Yong Wang,Ming Liu,Mingfeng Jiang,Rong Gao,Gang Wang | | Plasmid. 2019; : 102441 | | [Pubmed] | [DOI] | | | 51 |
Optimization and characterization of calcium phosphate transfection in mesenchymal stem cells |
|
| Chi-Wen Lo,Tzu-Hua Lin,Masaya Ueno,Monica Romero-Lopez,Masahiro Maruyama,Yusuke Kohno,Claire Rhee,Zhenyu Yao,Magdiel Pérez-Cruz,Everett Meyer,Stuart B. Goodman | | Tissue Engineering Part C: Methods. 2019; | | [Pubmed] | [DOI] | | | 52 |
CRISPR/Cas System for Genome Editing: Progress and Prospects as a Therapeutic Tool |
|
| Deepak Kumar Sahel,Anupama Mittal,Deepak Chitkara | | Journal of Pharmacology and Experimental Therapeutics. 2019; 370(3): 725 | | [Pubmed] | [DOI] | | | 53 |
Electrospinning Nanofibers for Therapeutics Delivery |
|
| S. Shahriar,Jagannath Mondal,Mohammad Hasan,Vishnu Revuri,Dong Lee,Yong-Kyu Lee | | Nanomaterials. 2019; 9(4): 532 | | [Pubmed] | [DOI] | | | 54 |
Creatine based polymer for codelivery of bioengineered MicroRNA and chemodrugs against breast cancer lung metastasis |
|
| Jieni Xu,Jingjing Sun,Pui Yan Ho,Zhangyi Luo,Weina Ma,Wenchen Zhao,Sanjay B. Rathod,Christian A. Fernandez,Raman Venkataramanan,Wen Xie,Ai-Ming Yu,Song Li | | Biomaterials. 2019; | | [Pubmed] | [DOI] | | | 55 |
Engineering Human Epidermal Growth Receptor 2-Targeting Hepatitis B Virus Core Nanoparticles for siRNA Delivery in Vitro and in Vivo |
|
| Izzat F. M. Suffian,Julie T.-W. Wang,Farid N. Faruqu,Julio Benitez,Yuya Nishimura,Chiaki Ogino,Akihiko Kondo,Khuloud T. Al-Jamal | | ACS Applied Nano Materials. 2018; | | [Pubmed] | [DOI] | | | 56 |
Multimeric Amphipathic a-Helical Sequences for Rapid and Efficient Intracellular Protein Transport at Nanomolar Concentrations |
|
| Jae Hoon Oh,Seung-Eun Chong,Sohee Nam,Soonsil Hyun,Sejong Choi,Hyojun Gye,Sangmok Jang,Joomyung Jang,Sung Won Hwang,Jaehoon Yu,Yan Lee | | Advanced Science. 2018; : 1800240 | | [Pubmed] | [DOI] | | | 57 |
Nucleic-acid based gene therapy approaches for sepsis |
|
| Yuichi Hattori,Kohshi Hattori,Tokiko Suzuki,Sailesh Palikhe,Naoyuki Matsuda | | European Journal of Pharmacology. 2018; | | [Pubmed] | [DOI] | | | 58 |
The method of chicken whole embryo culture using the eggshell windowing, surrogate eggshell and ex ovo culture system |
|
| M. Farzaneh,F. Attari,S. E. Khoshnam,P. E. Mozdziak | | British Poultry Science. 2018; : 1 | | [Pubmed] | [DOI] | | | 59 |
Therapeutic potential of combined viral transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases |
|
| Joshua Kuruvilla,Andrew Octavian Sasmita,Anna Pick Kiong Ling | | Neurological Sciences. 2018; | | [Pubmed] | [DOI] | | | 60 |
Enhanced transfection of a macromolecular lignin-based DNA complex with low cellular toxicity |
|
| Yoon Khei Ho,Dan Kai,Geraldine Xue En Tu,G. Roshan Deen,Heng Phon Too,Xian Jun Loh | | Bioscience Reports. 2018; 38(6) | | [Pubmed] | [DOI] | | | 61 |
Poly-l-lysine-coated superparamagnetic nanoparticles: a novel method for the transfection of pro-BDNF into neural stem cells |
|
| Salim Naama Albukhaty,Hossein Naderi-Manesh,Tiraihi Taqi,Majid Sakhi Jabir | | Artificial Cells, Nanomedicine, and Biotechnology. 2018; : 1 | | [Pubmed] | [DOI] | | | 62 |
A siRNA-induced peptide co-assembly system as a peptide-based siRNA nanocarrier for cancer therapy |
|
| Wenjun Li,Dongyuan Wang,Xiaodong Shi,Jingxu Li,Yue Ma,Yanding Wang,Tingting Li,Jianing Zhang,Rongtong Zhao,Zhiqiang Yu,Feng Yin,Zigang Li | | Materials Horizons. 2018; | | [Pubmed] | [DOI] | | | 63 |
Bombesin/oligoarginine fusion peptides for gastrin releasing peptide receptor (GRPR) targeted gene delivery |
|
| Anjuman Ara Begum,Yu Wan,Istvan Toth,Peter M. Moyle | | Bioorganic & Medicinal Chemistry. 2018; 26(2): 516 | | [Pubmed] | [DOI] | | | 64 |
Poloxamers, poloxamines and polymeric micelles: Definition, structure and therapeutic applications in cancer |
|
| Mauro Almeida,Mariana Magalhăes,Francisco Veiga,Ana Figueiras | | Journal of Polymer Research. 2018; 25(1) | | [Pubmed] | [DOI] | | | 65 |
Desarrollo de vectores génicos basados en polímeros sintéticos: PEI y PDMAEMA |
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| Ivonne Lorena Díaz-Ariza,César Augusto Sierra,León Darío Pérez-Pérez | | Revista Colombiana de Ciencias Químico-Farmacéuticas. 2018; 47(3): 350 | | [Pubmed] | [DOI] | | | 66 |
Hepatocellular targeted a-tocopherol based pH sensitive galactosylated lipids: design, synthesis and transfection studies |
|
| Venkanna Muripiti,Brijesh Lohchania,Srujan Kumar Marepally,Srilakshmi V. Patri | | MedChemComm. 2018; | | [Pubmed] | [DOI] | | | 67 |
Polyethylenimine-based nanocarriers in co-delivery of drug and gene: a developing horizon |
|
| Abbas Zakeri,Mohammad Amin Jadidi Kouhbanani,Nasrin Beheshtkhoo,Vahid Beigi,Seyyed Mojtaba Mousavi,Seyyed Ali Reza Hashemi,Ayoob Karimi Zade,Ali Mohammad Amani,Amir Savardashtaki,Esmail Mirzaei,Sara Jahandideh,Ahmad Movahedpour | | Nano Reviews & Experiments. 2018; 9(1): 1488497 | | [Pubmed] | [DOI] | | | 68 |
Live attenuated influenza vaccine viral vector induces functional cytotoxic T-cell immune response against foreign CD8+ T-cell epitopes inserted into NA and NS1 genes using the 2A self-cleavage site |
|
| Tatiana Kotomina,Daniil Korenkov,Victoria Matyushenko,Polina Prokopenko,Larisa Rudenko,Irina Isakova-Sivak | | Human Vaccines & Immunotherapeutics. 2018; | | [Pubmed] | [DOI] | | | 69 |
Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics |
|
| Graham McClorey,Subhashis Banerjee | | Biomedicines. 2018; 6(2): 51 | | [Pubmed] | [DOI] | | | 70 |
Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing |
|
| Yu Zhang,Chengzu Long,Rhonda Bassel-Duby,Eric N. Olson | | Physiological Reviews. 2018; 98(3): 1205 | | [Pubmed] | [DOI] | | | 71 |
Beyond gene transfection with methacrylate-based polyplexes – the influence of the amino substitution pattern |
|
| Ulrich S. Schubert,Tanja Bus,Martin Reifarth,Johannes C. Brendel,Stephanie Hoeppener,Anja Traeger,Anne-Kristin Trützschler | | Bioconjugate Chemistry. 2018; | | [Pubmed] | [DOI] | | | 72 |
Membrane permeabilizing amphiphilic peptide delivers recombinant transcription factor and CRISPR-Cas9/Cpf1 ribonucleoproteins in hard-to-modify cells |
|
| Thomas Del’Guidice,Jean-Pascal Lepetit-Stoffaes,Louis-Jean Bordeleau,Joannie Roberge,Vanessa Théberge,Coraline Lauvaux,Xavier Barbeau,Jessica Trottier,Vibhuti Dave,Denis-Claude Roy,Bruno Gaillet,Alain Garnier,David Guay,Alfred S Lewin | | PLOS ONE. 2018; 13(4): e0195558 | | [Pubmed] | [DOI] | | | 73 |
Gene-silencing suppressors for high-level production of the HIV-1 entry inhibitor griffithsin in Nicotiana benthamiana |
|
| Peyman Habibi,Carlos Ricardo Soccol,Barry R. O’Keefe,Lauren R.H. Krumpe,Jennifer Wilson,Leonardo Lima Pepino de Macedo,Muhammad Faheem,Vanessa Olinto Dos Santos,Guilherme Souza Prado,Marco Antonio Botelho,Severine Lacombe,Maria Fatima Grossi-de-Sa | | Process Biochemistry. 2018; | | [Pubmed] | [DOI] | | | 74 |
Mechanisms of unprimed and dexamethasone-primed nonviral gene delivery to human mesenchymal stem cells |
|
| Andrew Hamann,Kelly Broad,Albert Nguyen,Angela K. Pannier | | Biotechnology and Bioengineering. 2018; | | [Pubmed] | [DOI] | | | 75 |
Transfection efficiencies of a-tocopherylated cationic gemini lipids with hydroxyethyl bearing headgroups under high serum conditions |
|
| Bappa Maiti,Mohini Kamra,Anjali A. Karande,Santanu Bhattacharya | | Organic & Biomolecular Chemistry. 2018; | | [Pubmed] | [DOI] | | | 76 |
RNA Structure Design Improves Activity and Specificity of trans -Splicing-Triggered Cell Death in a Suicide Gene Therapy Approach |
|
| Sushmita Poddar,Pei She Loh,Zi Hao Ooi,Farhana Osman,Joachim Eul,Volker Patzel | | Molecular Therapy - Nucleic Acids. 2018; 11: 41 | | [Pubmed] | [DOI] | | | 77 |
Cell surface engineering and application in cell delivery to heart diseases |
|
| Daniel Y. Lee,Byung-Hyun Cha,Minjin Jung,Angela S. Kim,David A. Bull,Young-Wook Won | | Journal of Biological Engineering. 2018; 12(1) | | [Pubmed] | [DOI] | | | 78 |
Engineering DNA vaccines against infectious diseases |
|
| Jihui Lee,Shreedevi Arun Kumar,Yong Yu Jhan,Corey J. Bishop | | Acta Biomaterialia. 2018; | | [Pubmed] | [DOI] | | | 79 |
A Universal GSH-Responsive Nanoplatform for the Delivery of DNA, mRNA, and Cas9/sgRNA Ribonucleoprotein |
|
| Guojun Chen,Ben Ma,Yuyuan Wang,Shaoqin Gong | | ACS Applied Materials & Interfaces. 2018; | | [Pubmed] | [DOI] | | | 80 |
Crustacean nuclear localization signals help facilitating the delivery of DNA into Australian red-claw crayfish cells |
|
| Chan D.H. Nguyen,Tomer Ventura,Abigail Elizur | | Aquaculture. 2018; | | [Pubmed] | [DOI] | | | 81 |
Roles of microRNAs in T cell immunity: Implications for strategy development against infectious diseases |
|
| Bikash R. Giri,Ram I. Mahato,Guofeng Cheng | | Medicinal Research Reviews. 2018; | | [Pubmed] | [DOI] | | | 82 |
Design of PEI-conjugated bio-reducible polymer for efficient gene delivery |
|
| Joung-Pyo Nam,Soyoung Kim,Sung Wan Kim | | International Journal of Pharmaceutics. 2018; | | [Pubmed] | [DOI] | | | 83 |
Chitosan in Non-Viral Gene Delivery: Role of Structure, Characterization Methods, and Insights in Cancer and Rare Diseases Therapies |
|
| Beatriz Santos-Carballal,Elena Fernández Fernández,Francisco Goycoolea | | Polymers. 2018; 10(4): 444 | | [Pubmed] | [DOI] | | | 84 |
Comparison of random and gradient amino functionalized poly(2-oxazoline)s: Can the transfection efficiency be tuned by the macromolecular structure? |
|
| David Hertz,Meike N. Leiske,Thomas Wloka,Anja Traeger,Matthias Hartlieb,Michael M. Kessels,Stephanie Schubert,Britta Qualmann,Ulrich S. Schubert | | Journal of Polymer Science Part A: Polymer Chemistry. 2018; | | [Pubmed] | [DOI] | | | 85 |
In vivo methods for acute modulation of gene expression in the central nervous system |
|
| Andrzej W. Cwetsch,Bruno Pinto,Annalisa Savardi,Laura Cancedda | | Progress in Neurobiology. 2018; | | [Pubmed] | [DOI] | | | 86 |
Electrostatically assembled dendrimer complex with a high-affinity protein binder for targeted gene delivery |
|
| Jong-won Kim,Joong-jae Lee,Joon Sig Choi,Hak-Sung Kim | | International Journal of Pharmaceutics. 2018; 544(1): 39 | | [Pubmed] | [DOI] | | | 87 |
PEGylated Chitosan for Nonviral Aerosol and Mucosal Delivery of the CRISPR/Cas9 System in Vitro |
|
| Hairui Zhang,Tania F. Bahamondez-Canas,Yajie Zhang,Jasmim Leal,Hugh D.C. Smyth | | Molecular Pharmaceutics. 2018; | | [Pubmed] | [DOI] | | | 88 |
Recombinant histones as an instrument for delivery of nucleic acids into eukaryotic cells |
|
| M. V. Zinovyeva,A. V. Sass,A. V. Vvedensky,V. K. Potapov,L. G. Nikolaev,E. D. Sverdlov | | Molecular Genetics Microbiology and Virology (Russian version). 2018; 36(3): 30 | | [Pubmed] | [DOI] | | | 89 |
Structure-activity relationship of serotonin derived tocopherol lipids |
|
| Venkanna Muripiti,Thasneem Yoosuf Mujahid,Venkata Harsha Vardhan Boddeda,Shrish Tiwari,Srujan Kumar Marepally,Srilakshmi V Patri,Vijaya Gopal | | International Journal of Pharmaceutics. 2018; | | [Pubmed] | [DOI] | | | 90 |
Efficient Nonviral Transfection of Human Bone Marrow Mesenchymal Stromal Cells Shown Using Placental Growth Factor Overexpression |
|
| Winston Y. Cheung,Owen Hovey,Jonathan M. Gobin,Gauri Muradia,Jelica Mehic,Carole Westwood,Jessie R. Lavoie | | Stem Cells International. 2018; 2018: 1 | | [Pubmed] | [DOI] | | | 91 |
Recent advances in stem cell therapeutics and tissue engineering strategies |
|
| Seong Gyu Kwon,Yang Woo Kwon,Tae Wook Lee,Gyu Tae Park,Jae Ho Kim | | Biomaterials Research. 2018; 22(1) | | [Pubmed] | [DOI] | | | 92 |
Intravenous Delivery of piggyBac Transposons as a Useful Tool for Liver-Specific Gene-Switching |
|
| Shingo Nakamura,Masayuki Ishihara,Satoshi Watanabe,Naoko Ando,Masato Ohtsuka,Masahiro Sato | | International Journal of Molecular Sciences. 2018; 19(11): 3452 | | [Pubmed] | [DOI] | | | 93 |
Current viral-mediated gene transfer research for treatment of Alzheimer’s disease |
|
| Andrew Octavian Sasmita | | Biotechnology and Genetic Engineering Reviews. 2018; : 1 | | [Pubmed] | [DOI] | | | 94 |
Recombinant Histones as an Instrument for the Delivery of Nucleic Acids into Eukaryotic Cells |
|
| M. V. Zinovyeva,A. V. Sass,A. V. Vvedensky,V. K. Potapov,L. G. Nikolaev,E. D. Sverdlov | | Molecular Genetics, Microbiology and Virology. 2018; 33(3): 187 | | [Pubmed] | [DOI] | | | 95 |
Inhalable particulate drug delivery systems for lung cancer therapy: Nanoparticles, microparticles, nanocomposites and nanoaggregates |
|
| Hadeer M. Abdelaziz,Mohamed Gaber,Mahmoud M. Abd-Elwakil,Moustafa T. Mabrouk,Mayada M. Elgohary,Nayra M. Kamel,Dalia M. Kabary,May S. Freag,Magda W. Samaha,Sana M. Mortada,Kadria A. Elkhodairy,Jia-You Fang,Ahmed O. Elzoghby | | Journal of Controlled Release. 2018; 269: 374 | | [Pubmed] | [DOI] | | | 96 |
Preparation of gene drug delivery systems of Cationic peptide lipid with 0G-PAMAM as hydrophilic end and its biological properties evaluation |
|
| Yingying Feng,Hu Haimei,Shuanghong liang,Dan Wang | | Chemistry and Physics of Lipids. 2018; | | [Pubmed] | [DOI] | | | 97 |
Gene therapy and type 1 diabetes mellitus |
|
| Dinesh Kumar Chellappan,Nandhini S. Sivam,Kai Xiang Teoh,Wai Pan Leong,Tai Zhen Fui,Kien Chooi,Nico Khoo,Fam Jia Yi,Jestin Chellian,Lim Lay Cheng,Rajiv Dahiya,Gaurav Gupta,Gautam Singhvi,Srinivas Nammi,Philip Michael Hansbro,Kamal Dua | | Biomedicine & Pharmacotherapy. 2018; 108: 1188 | | [Pubmed] | [DOI] | | | 98 |
Acylation of the S413-PV cell-penetrating peptide as a means of enhancing its capacity to mediate nucleic acid delivery: Relevance of peptide/lipid interactions |
|
| Catarina M. Morais,Ana M. Cardoso,Pedro P. Cunha,Luísa Aguiar,Nuno Vale,Emílio Lage,Marina Pinheiro,Cláudia Nunes,Paula Gomes,Salette Reis,M. Margarida C.A. Castro,Maria C. Pedroso de Lima,Amália S. Jurado | | Biochimica et Biophysica Acta (BBA) - Biomembranes. 2018; 1860(12): 2619 | | [Pubmed] | [DOI] | | | 99 |
The determination of gemini surfactants used as gene delivery agents in cellular matrix using validated tandem mass spectrometric method |
|
| Jin Wei,Badea Ildiko,C. Leary Scot,El-need Anas | | Journal of Pharmaceutical and Biomedical Analysis. 2018; | | [Pubmed] | [DOI] | | | 100 |
Enhanced gene transfection efficiency by low-dose 25?kDa polyethylenimine by the assistance of 1.8?kDa polyethylenimine |
|
| Hui Zhang,Zhiyi Chen,Meng Du,Yue Li,Yuhao Chen | | Drug Delivery. 2018; 25(1): 1740 | | [Pubmed] | [DOI] | | | 101 |
Evaluating Nonintegrating Lentiviruses as Safe Vectors for Noninvasive Reporter-Based Molecular Imaging of Multipotent Mesenchymal Stem Cells |
|
| Amanda M. Hamilton,Paula J. Foster,John A. Ronald | | Human Gene Therapy. 2018; 29(10): 1213 | | [Pubmed] | [DOI] | | | 102 |
Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles |
|
| Marianna Yanez Arteta,Tomas Kjellman,Stefano Bartesaghi,Simonetta Wallin,Xiaoqiu Wu,Alexander J. Kvist,Aleksandra Dabkowska,Noémi Székely,Aurel Radulescu,Johan Bergenholtz,Lennart Lindfors | | Proceedings of the National Academy of Sciences. 2018; : 201720542 | | [Pubmed] | [DOI] | | | 103 |
Exosomes: natural nanoparticles as bio shuttles for RNAi delivery |
|
| Saber Ghazizadeh Darband,Mohammad Mirza-Aghazadeh-Attari,Mojtaba Kaviani,Ainaz Mihanfar,Shirin Sadighparvar,Bahman Yousefi,Maryam Majidinia | | Journal of Controlled Release. 2018; 289: 158 | | [Pubmed] | [DOI] | | | 104 |
BMP2 expressing genetically engineered mesenchymal stem cells on composite fibrous scaffolds for enhanced bone regeneration in segmental defects |
|
| Shruthy Kuttappan,A. Anitha,M.G. Minsha,Parvathy M. Menon,T.B. Sivanarayanan,Lakshmi Sumitra Vijayachandran,Manitha B. Nair | | Materials Science and Engineering: C. 2018; | | [Pubmed] | [DOI] | | | 105 |
Engineering and cytosolic delivery of a native regulatory protein and its variants for modulation of ERK2 signaling pathway |
|
| Jeong-Hyun Ryou,Yoo-Kyoung Sohn,Dong-Gun Kim,Hyun-Ho Kyeong,Hak-Sung Kim | | Biotechnology and Bioengineering. 2018; | | [Pubmed] | [DOI] | | | 106 |
Rational Design of a siRNA Delivery System: ALOX5 and Cancer Stem Cells as Therapeutic Targets |
|
| Diana Rafael,Fernanda Andrade,Sara Montero,Petra Gener,Joaquin Seras-Franzoso,Francesc Martínez,Patricia González,Helena Florindo,Diego Arango,Joan Sayós,Ibane Abasolo,Mafalda Videira,Simó Schwartz Jr. | | Precision Nanomedicine. 2018; 1(2): 86 | | [Pubmed] | [DOI] | | | 107 |
Modulating angiogenesis with integrin-targeted nanomedicines |
|
| Aroa Duro-Castano,Elena Gallon,Caitlin Decker,María J. Vicent | | Advanced Drug Delivery Reviews. 2017; | | [Pubmed] | [DOI] | | | 108 |
Bioengineered liposome–scaffold composites as therapeutic delivery systems |
|
| Claudia Zylberberg,Sandro Matosevic | | Therapeutic Delivery. 2017; 8(6): 425 | | [Pubmed] | [DOI] | | | 109 |
A mechanistic investigation exploring the differential transfection efficiencies between the easy-to-transfect SK-BR3 and difficult-to-transfect CT26 cell lines |
|
| Elizabeth Figueroa,Pallavi Bugga,Vishwaratn Asthana,Allen L. Chen,J. Stephen Yan,Emily Reiser Evans,Rebekah A. Drezek | | Journal of Nanobiotechnology. 2017; 15(1) | | [Pubmed] | [DOI] | | | 110 |
Pharmacological Reprogramming of Somatic Cells for Regenerative Medicine |
|
| Min Xie,Shibing Tang,Ke Li,Sheng Ding | | Accounts of Chemical Research. 2017; | | [Pubmed] | [DOI] | | | 111 |
Modifying plasmid-loaded HSA-nanoparticles with cell penetrating peptides – Cellular uptake and enhanced gene delivery |
|
| J. Mesken,A. Iltzsche,D. Mulac,K. Langer | | International Journal of Pharmaceutics. 2017; 522(1-2): 198 | | [Pubmed] | [DOI] | | | 112 |
The evolution of heart failure with reduced ejection fraction pharmacotherapy: What do we have and where are we going? |
|
| Ahmed Selim,Ronald Zolty,Yiannis S. Chatzizisis | | Pharmacology & Therapeutics. 2017; | | [Pubmed] | [DOI] | | | 113 |
Polymeric nanoparticles as cancer-specific DNA delivery vectors to human hepatocellular carcinoma |
|
| Camila G. Zamboni,Kristen L. Kozielski,Hannah J. Vaughan,Maisa M. Nakata,Jayoung Kim,Luke J. Higgins,Martin G. Pomper,Jordan J. Green | | Journal of Controlled Release. 2017; | | [Pubmed] | [DOI] | | | 114 |
Generation and Characterization of Virus-Enhancing Peptide Nanofibrils Functionalized with Fluorescent Labels |
|
| Sascha Rode,Manuel Hayn,Annika Röcker,Stefanie Sieste,Markus Lamla,Daniel Markx,Christoph Meier,Frank Kirchhoff,Paul Walther,Marcus Fändrich,Tanja Weil,Jan Münch | | Bioconjugate Chemistry. 2017; 28(4): 1260 | | [Pubmed] | [DOI] | | | 115 |
Retro-inverso d -peptide-modified hyaluronic acid/bioreducible hyperbranched poly(amido amine)/pDNA core-shell ternary nanoparticles for the dual-targeted delivery of short hairpin RNA-encoding plasmids |
|
| Jijin Gu,Xinyi Chen,Xiaoling Fang,Xianyi Sha | | Acta Biomaterialia. 2017; | | [Pubmed] | [DOI] | | | 116 |
Gene Therapy for Pancreatic Cancer: Specificity, Issues and Hopes |
|
| Marie Rouanet,Marine Lebrin,Fabian Gross,Barbara Bournet,Pierre Cordelier,Louis Buscail | | International Journal of Molecular Sciences. 2017; 18(6): 1231 | | [Pubmed] | [DOI] | | | 117 |
Stimuli-Regulated Smart Polymeric Systems for Gene Therapy |
|
| Ansuja Mathew,Ki-Hyun Cho,Saji Uthaman,Chong-Su Cho,In-Kyu Park | | Polymers. 2017; 9(4): 152 | | [Pubmed] | [DOI] | | | 118 |
Genetically modified mesenchymal stem/stromal cells transfected with adiponectin gene can stably secrete adiponectin |
|
| Md. Murad Hossain,Malliga Raman Murali,Tunku Kamarul | | Life Sciences. 2017; 182: 50 | | [Pubmed] | [DOI] | | | 119 |
Adenovirus-mediated gene delivery: Potential applications for gene and cell-based therapies in the new era of personalized medicine |
|
| Cody S. Lee,Elliot S. Bishop,Ruyi Zhang,Xinyi Yu,Evan M. Farina,Shujuan Yan,Chen Zhao,Zongyue Zeng,Yi Shu,Xingye Wu,Jiayan Lei,Yasha Li,Wenwen Zhang,Chao Yang,Ke Wu,Ying Wu,Sherwin Ho,Aravind Athiviraham,Michael J. Lee,Jennifer Moriatis Wolf,Russell R. Reid,Tong-Chuan He | | Genes & Diseases. 2017; 4(2): 43 | | [Pubmed] | [DOI] | | | 120 |
Interactions between microRNAs and long non-coding RNAs in cardiac development and repair |
|
| Alessio Rotini,Ester Martínez-Sarrŕ,Enrico Pozzo,Maurilio Sampaolesi | | Pharmacological Research. 2017; | | [Pubmed] | [DOI] | | | 121 |
Evolution of New “Bolaliposomes” using Novel a-Tocopheryl Succinate Based Cationic Lipid and 1,12-Disubstituted Dodecane-Based Bolaamphiphile for Efficient Gene Delivery |
|
| Mallikarjun Gosangi,Hithavani Rapaka,Venkatesh Ravula,Srilakshmi V. Patri | | Bioconjugate Chemistry. 2017; | | [Pubmed] | [DOI] | | | 122 |
Boron nitride nanotubes for gene silencing |
|
| Özlem Sen,Zehra Çobandede,Melis Emanet,Ömer Faruk Bayrak,Mustafa Çulha | | Biochimica et Biophysica Acta (BBA) - General Subjects. 2017; | | [Pubmed] | [DOI] | | | 123 |
Multifaceted Applications of Chitosan in Cancer Drug Delivery and Therapy |
|
| Anish Babu,Rajagopal Ramesh | | Marine Drugs. 2017; 15(4): 96 | | [Pubmed] | [DOI] | | | 124 |
Polyamidoamine (PAMAM) Dendrimers Modified with Cathepsin-B Cleavable Oligopeptides for Enhanced Gene Delivery |
|
| Seulgi Lee,Sang Son,Su Song,Tai Ha,Joon Choi | | Polymers. 2017; 9(6): 224 | | [Pubmed] | [DOI] | | | 125 |
Synthesis and Degradation Study of Cationic Polycaprolactone-Based Nanoparticles for Biomedical and Industrial Applications |
|
| Azzurra Agostini,Simone Gatti,Alberto Cesana,Davide Moscatelli | | Industrial & Engineering Chemistry Research. 2017; 56(20): 5872 | | [Pubmed] | [DOI] | | | 126 |
Glycosaminoglycan and Proteoglycan-Based Biomaterials: Current Trends and Future Perspectives |
|
| Jelena Rnjak-Kovacina,Fengying Tang,John M. Whitelock,Megan S. Lord | | Advanced Healthcare Materials. 2017; : 1701042 | | [Pubmed] | [DOI] | | | 127 |
Molecular Communication and Nanonetwork for Targeted Drug Delivery: A Survey |
|
| Uche A. K. Chude-Okonkwo,Reza Malekian,B. T. Maharaj,Athanasios V. Vasilakos | | IEEE Communications Surveys & Tutorials. 2017; 19(4): 3046 | | [Pubmed] | [DOI] | | | 128 |
Mesenchymal stem cells: A new platform for targeting suicide genes in cancer |
|
| Rana Moradian Tehrani,Javad Verdi,Mahdi Noureddini,Rasoul Salehi,Reza Salarinia,Meysam Mosalaei,Miganosh Simonian,Behrang Alani,Moosa Rahimi Ghiasi,Mahmoud Reza Jaafari,Hamed Reza Mirzaei,Hamed Mirzaei | | Journal of Cellular Physiology. 2017; | | [Pubmed] | [DOI] | | | 129 |
The use of nanoparticulates to treat breast cancer |
|
| Xiaomeng Tang,Welley S Loc,Cheng Dong,Gail L Matters,Peter J Butler,Mark Kester,Craig Meyers,Yixing Jiang,James H Adair | | Nanomedicine. 2017; | | [Pubmed] | [DOI] | | | 130 |
Technical Improvement and Application of Hydrodynamic Gene Delivery in Study of Liver Diseases |
|
| Mei Huang,Rui Sun,Qiang Huang,Zhigang Tian | | Frontiers in Pharmacology. 2017; 8 | | [Pubmed] | [DOI] | | | 131 |
Advances in the design of solid lipid nanoparticles and nanostructured lipid carriers for targeting brain diseases |
|
| Christos Tapeinos,Matteo Battaglini,Gianni Ciofani | | Journal of Controlled Release. 2017; | | [Pubmed] | [DOI] | | | 132 |
Block copolymer conjugated Au-coated Fe3O4 nanoparticles as vectors for enhancing colloidal stability and cellular uptake |
|
| Junbo Li,Sheng Zou,Jiayu Gao,Ju Liang,Huiyun Zhou,Lijuan Liang,Wenlan Wu | | Journal of Nanobiotechnology. 2017; 15(1) | | [Pubmed] | [DOI] | | | 133 |
Exosome-based small RNA delivery: progress and prospects |
|
| Mei Lu,Haonan Xing,Zhe Xun,Tianzhi Yang,Pingtian Ding,Cuifang Cai,Dongkai Wang,Xiaoyun Zhao | | Asian Journal of Pharmaceutical Sciences. 2017; | | [Pubmed] | [DOI] | | | 134 |
Engineering brown fat into skeletal muscle using ultrasound-targeted microbubble destruction gene delivery in obese Zucker rats: Proof of concept design |
|
| Raul A. Bastarrachea,Jiaxi Chen,Jack W. Kent,Edna J. Nava-Gonzalez,Ernesto Rodriguez-Ayala,Marcel M. Daadi,Barbara Jorge,Hugo Laviada-Molina,Anthony G. Comuzzie,Shuyuan Chen,Paul A. Grayburn | | IUBMB Life. 2017; | | [Pubmed] | [DOI] | | | 135 |
Green Transfection: Cationic Lipid Nanocarrier System Derivatized from Vegetable Fat, Palmstearin Enhances Nucleic Acid Transfections |
|
| Priya Dharmalingam,Hari Krishna R. Rachamalla,Brijesh Lohchania,Bhanuprasad Bandlamudi,Saravanabhavan Thangavel,Mohankumar K. Murugesan,Rajkumar Banerjee,Arabinda Chaudhuri,Chandrashekhar Voshavar,Srujan Marepally | | ACS Omega. 2017; 2(11): 7892 | | [Pubmed] | [DOI] | | | 136 |
Honing Cell and Tissue Culture Conditions for Bone and Cartilage Tissue Engineering |
|
| Johnny Lam,Esther J. Lee,Elisa C. Clark,Antonios G. Mikos | | Cold Spring Harbor Perspectives in Medicine. 2017; 7(12): a025734 | | [Pubmed] | [DOI] | | | 137 |
Amphiphilic small peptides for delivery of plasmid DNAs and siRNAs |
|
| Eun-Kyoung Bang,Hanna Cho,Sean S.-H. Jeon,Na Ly Tran,Dong-Kwon Lim,Wooyoung Hur,Taebo Sim | | Chemical Biology & Drug Design. 2017; | | [Pubmed] | [DOI] | | | 138 |
Method for Dual Viral Vector Mediated CRISPR-Cas9 Gene Disruption in Primary Human Endothelial Cells |
|
| Haixia Gong,Menglin Liu,Jeff Klomp,Bradley J. Merrill,Jalees Rehman,Asrar B. Malik | | Scientific Reports. 2017; 7: 42127 | | [Pubmed] | [DOI] | | | 139 |
Concise Review: The Potential Use of Intestinal Stem Cells to Treat Patients with Intestinal Failure |
|
| Sung Noh Hong,James C.Y. Dunn,Matthias Stelzner,Martín G. Martín | | STEM CELLS Translational Medicine. 2017; 6(2): 666 | | [Pubmed] | [DOI] | | | 140 |
Hydrodynamic gene delivery in human skin using a hollow microneedle device |
|
| M Dul,M Stefanidou,P Porta,J Serve,C OćMahony,B Malissen,S Henri,Y Levin,E Kochba,FS Wong,C Dayan,SA Coulman,JC Birchall | | Journal of Controlled Release. 2017; | | [Pubmed] | [DOI] | | | 141 |
Acceleration of bone regeneration in bioactive glass/gelatin composite scaffolds seeded with bone marrow-derived mesenchymal stem cells over-expressing bone morphogenetic protein-7 |
|
| Saeid Kargozar,Seyed Jafar Hashemian,Mansooreh Soleimani,Peiman Brouki Milan,Mohammad Askari,Vahid Khalaj,Ali Samadikuchaksaraie,Sepideh Hamzehlou,Amir Reza Katebi,Noorahmad Latifi,Masoud Mozafari,Francesco Baino | | Materials Science and Engineering: C. 2017; 75: 688 | | [Pubmed] | [DOI] | | | 142 |
Ternary complex of plasmid DNA with NLS-Mu-Mu protein and cationic niosome for biocompatible and efficient gene delivery: a comparative study with protamine and lipofectamine |
|
| Mohammad Hadi Nematollahi,Masoud Torkzadeh-Mahanai,Abbas Pardakhty,Hossein Ali Ebrahimi Meimand,Gholamreza Asadikaram | | Artificial Cells, Nanomedicine, and Biotechnology. 2017; : 1 | | [Pubmed] | [DOI] | | | 143 |
Insights into the key roles of epigenetics in matrix macromolecules-associated wound healing |
|
| Zoi Piperigkou,Martin Götte,Achilleas D. Theocharis,Nikos K. Karamanos | | Advanced Drug Delivery Reviews. 2017; | | [Pubmed] | [DOI] | | | 144 |
Synthesis of magnetic cytosine-imprinted chitosan nanoparticles |
|
| Mei-Hwa Lee,Arti Ahluwalia,Jian-Zhou Chen,Neng-Lang Shih,Hung-Yin Lin | | Nanotechnology. 2017; 28(8): 085705 | | [Pubmed] | [DOI] | | | 145 |
Gene therapies development: slow progress and promising prospect |
|
| Eve Hanna,Cécile Rémuzat,Pascal Auquier,Mondher Toumi | | Journal of Market Access & Health Policy. 2017; 5(1): 1265293 | | [Pubmed] | [DOI] | | | 146 |
Integrated microfluidic devices for the synthesis of nanoscale liposomes and lipoplexes |
|
| Tiago A. Balbino,Juliana M. Serafin,Allan Radaic,Marcelo B. de Jesus,Lucimara G. de la Torre | | Colloids and Surfaces B: Biointerfaces. 2017; | | [Pubmed] | [DOI] | | | 147 |
Fluorescent nanoswitch for monitoring specific pluripotency-related microRNAs of induced pluripotent stem cells: Development of polyethyleneimineoligonucleotide hybridization probes |
|
| Seungmin Han,Hye Young Son,Byunghoon Kang,Eunji Jang,Jisun Ki,Na Geum Lee,Jongjin Park,Moo-Kwang Shin,Byeonggeol Mun,Jeong-Ki Min,Seungjoo Haam | | Nano Research. 2017; | | [Pubmed] | [DOI] | | | 148 |
Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System |
|
| Diego Pignataro,Diego Sucunza,Lucia Vanrell,Esperanza Lopez-Franco,Iria G. Dopeso-Reyes,Africa Vales,Mirja Hommel,Alberto J. Rico,Jose L. Lanciego,Gloria Gonzalez-Aseguinolaza | | Frontiers in Neuroanatomy. 2017; 11 | | [Pubmed] | [DOI] | | | 149 |
Basic concepts and recent advances in nanogels as carriers for medical applications |
|
| Iordana Neamtu,Alina Gabriela Rusu,Alina Diaconu,Loredana Elena Nita,Aurica P. Chiriac | | Drug Delivery. 2017; 24(1): 539 | | [Pubmed] | [DOI] | | | 150 |
Transfection Studies with Colloidal Systems Containing Highly Purified Bipolar Tetraether Lipids from Sulfolobus acidocaldarius |
|
| Konrad H. Engelhardt,Shashank Reddy Pinnapireddy,Elias Baghdan,Jarmila Jedelská,Udo Bakowsky | | Archaea. 2017; 2017: 1 | | [Pubmed] | [DOI] | | | 151 |
Surfactant effect on the physicochemical characteristics of cationic solid lipid nanoparticles |
|
| Chiara Botto,Nicolň Mauro,Erika Amore,Elisabetta Martorana,Gaetano Giammona,Maria Luisa Bondě | | International Journal of Pharmaceutics. 2017; 516(1-2): 334 | | [Pubmed] | [DOI] | | | 152 |
Improved formulation of cationic solid lipid nanoparticles displays cellular uptake and biological activity of nucleic acids |
|
| Anna Fŕbregas,Silvia Prieto-Sánchez,Marc Suńé-Pou,Sofía Boyero-Corral,Josep Ramón Ticó,Encarna García-Montoya,Pilar Pérez-Lozano,Montserrat Mińarro,Josep MŞ Suńé-Negre,Cristina Hernández-Munain,Carlos Suńé | | International Journal of Pharmaceutics. 2017; 516(1-2): 39 | | [Pubmed] | [DOI] | | | 153 |
Combining Gene and Stem Cell Therapy for Peripheral Nerve Tissue Engineering |
|
| Francesca Busuttil,Ahad A. Rahim,James B. Phillips | | Stem Cells and Development. 2017; | | [Pubmed] | [DOI] | | | 154 |
Magnetic nanoparticles-based drug and gene delivery systems for the treatment of pulmonary diseases |
|
| Ibrahim M El-Sherbiny,Nancy M Elbaz,Mohammed Sedki,Abdulaziz Elgammal,Magdi H Yacoub | | Nanomedicine. 2017; | | [Pubmed] | [DOI] | | | 155 |
Composite liposome-PEI/nucleic acid lipopolyplexes for safe and efficient gene delivery and gene knockdown |
|
| Shashank Reddy Pinnapireddy,Lili Duse,Boris Strehlow,Jens Schäfer,Udo Bakowsky | | Colloids and Surfaces B: Biointerfaces. 2017; | | [Pubmed] | [DOI] | | | 156 |
Mesenchymal Stromal/Stem Cells: A New Era in the Cell-Based Targeted Gene Therapy of Cancer |
|
| Faroogh Marofi,Ghasem Vahedi,Alireza Biglari,Abdolreza Esmaeilzadeh,Seyyed Shamsadin Athari | | Frontiers in Immunology. 2017; 8 | | [Pubmed] | [DOI] | | | 157 |
Targeting renal fibrosis: Mechanisms and drug delivery systems |
|
| Madalina V. Nastase,Jinyang Zeng-Brouwers,Malgorzata Wygrecka,Liliana Schaefer | | Advanced Drug Delivery Reviews. 2017; | | [Pubmed] | [DOI] | | | 158 |
Freezing-Assisted Gene Delivery Combined with Polyampholyte Nanocarriers |
|
| Sana Ahmed,Tadashi Nakaji-Hirabayashi,Takayoshi Watanabe,Takahiro Hohsaka,Kazuaki Matsumura | | ACS Biomaterials Science & Engineering. 2017; | | [Pubmed] | [DOI] | | | 159 |
Recent advances on extracellular vesicles in therapeutic delivery: challenges, solutions, and opportunities |
|
| Mei Lu,Haonan Xing,Zhen Yang,Yanping Sun,Tianzhi Yang,Xiaoyun Zhao,Cuifang Cai,Dongkai Wang,Pingtian Ding | | European Journal of Pharmaceutics and Biopharmaceutics. 2017; | | [Pubmed] | [DOI] | | | 160 |
Functionalized 2D nanomaterials for gene delivery applications |
|
| Feng Yin,Bobo Gu,Yining Lin,Nishtha Panwar,Swee Chuan Tjin,Junle Qu,Shu Ping Lau,Ken-Tye Yong | | Coordination Chemistry Reviews. 2017; 347: 77 | | [Pubmed] | [DOI] | | | 161 |
Plant viral and bacteriophage delivery of nucleic acid therapeutics |
|
| Patricia Lam,Nicole F. Steinmetz | | Wiley Interdisciplinary Reviews: Nanomedicine and Nanobiotechnology. 2017; : e1487 | | [Pubmed] | [DOI] | | | 162 |
Functional therapies for cutaneous wound repair in epidermolysis bullosa |
|
| Patricia Peking,Ulrich Koller,Eva M. Murauer | | Advanced Drug Delivery Reviews. 2017; | | [Pubmed] | [DOI] | | | 163 |
Sustained viral gene delivery from a micro-fibrous, elastomeric cardiac patch to the ischemic rat heart |
|
| Xinzhu Gu,Yasumoto Matsumura,Ying Tang,Souvik Roy,Richard Hoff,Bing Wang,William R. Wagner | | Biomaterials. 2017; 133: 132 | | [Pubmed] | [DOI] | | | 164 |
Polyplex Evolution: Understanding Biology, Optimizing Performance |
|
| Arnaldur Hall,Ulrich Lächelt,Jiri Bartek,Ernst Wagner,Seyed Moein Moghimi | | Molecular Therapy. 2017; | | [Pubmed] | [DOI] | | | 165 |
Cationic ß-Cyclodextrin–Chitosan Conjugates as Potential Carrier for pmCherry-C1 Gene Delivery |
|
| Touba Eslaminejad,Seyed Noureddin Nematollahi-Mahani,Mehdi Ansari | | Molecular Biotechnology. 2016; 58(4): 287 | | [Pubmed] | [DOI] | | | 166 |
Systematically probing the bottom-up synthesis of AuPAMAM conjugates for enhanced transfection efficiency |
|
| Elizabeth R. Figueroa,J. Stephen Yan,Nicolette K. Chamberlain-Simon,Adam Y. Lin,Aaron E. Foster,Rebekah A. Drezek | | Journal of Nanobiotechnology. 2016; 14(1) | | [Pubmed] | [DOI] | | | 167 |
Gene delivery using calcium phosphate nanoparticles: Optimization of the transfection process and the effects of citrate and poly(l-lysine) as additives |
|
| Mohammed A. Khan,Victoria M. Wu,Shreya Ghosh,Vuk Uskokovic | | Journal of Colloid and Interface Science. 2016; 471: 48 | | [Pubmed] | [DOI] | | | 168 |
The promising alliance of anti-cancer electrochemotherapy with immunotherapy |
|
| Christophe Y. Calvet,Lluis M. Mir | | Cancer and Metastasis Reviews. 2016; | | [Pubmed] | [DOI] | | | 169 |
Graphene-based materials for tissue engineering |
|
| Su Ryon Shin,Yi-Chen Li,HaeLin Jang,Parastoo Khoshakhlagh,Mohsen Akbari,Amir Nasajpour,Yu Shrike Zhang,Ali Tamayol,Ali Khademhosseini | | Advanced Drug Delivery Reviews. 2016; | | [Pubmed] | [DOI] | | | 170 |
Vascular Repair by Circumferential Cell Therapy Using Magnetic Nanoparticles and Tailored Magnets |
|
| Sarah Vosen,Sarah Rieck,Alexandra Heidsieck,Olga Mykhaylyk,Katrin Zimmermann,Wilhelm Bloch,Dietmar Eberbeck,Christian Plank,Bernhard Gleich,Alexander Pfeifer,Bernd K. Fleischmann,Daniela Wenzel | | ACS Nano. 2016; 10(1): 369 | | [Pubmed] | [DOI] | | | 171 |
Epigenome Editing: State of the Art, Concepts, and Perspectives |
|
| Goran Kungulovski,Albert Jeltsch | | Trends in Genetics. 2016; 32(2): 101 | | [Pubmed] | [DOI] | | | 172 |
Nucleic Acid Delivery for Endothelial Dysfunction in Cardiovascular Diseases |
|
| Dipti Deshpande,David R. Janero,Victor Segura-Ibarra,Elvin Blanco,Mansoor M. Amiji | | Methodist DeBakey Cardiovascular Journal. 2016; 12(3): 134 | | [Pubmed] | [DOI] | | | 173 |
Functional lipids based on [12]aneN3and naphthalimide as efficient non-viral gene vectors |
|
| Yong-Guang Gao,Uzair Alam,Quan Tang,You-Di Shi,Ying Zhang,Ruibing Wang,Zhong-Lin Lu | | Org. Biomol. Chem.. 2016; 14(26): 6346 | | [Pubmed] | [DOI] | | | 174 |
Conjugates of small targeting molecules to non-viral vectors for the mediation of siRNA |
|
| Defu Zhi,Yinan Zhao,Shaohui Cui,Huiying Chen,Shubiao Zhang | | Acta Biomaterialia. 2016; 36: 21 | | [Pubmed] | [DOI] | | | 175 |
Significance of stem cell marker Nanog gene in the diagnosis and prognosis of lung cancer |
|
| Zeng Liu,Jing Zhang,Honggang Kang,Guiming Sun,Baozhong Wang,Yanwen Wang,Mengxiang Yang | | Oncology Letters. 2016; 12(4): 2507 | | [Pubmed] | [DOI] | | | 176 |
Genetic Modification of the Lung Directed Toward Treatment of Human Disease |
|
| Dolan Sondhi,Katie Stiles,Bishnu P De,Ronald G Crystal | | Human Gene Therapy. 2016; | | [Pubmed] | [DOI] | | | 177 |
Effect of sustained PDGF nonviral gene delivery on repair of tooth-supporting bone defects |
|
| A B Plonka,B Khorsand,N Yu,J V Sugai,A K Salem,W V Giannobile,S Elangovan | | Gene Therapy. 2016; | | [Pubmed] | [DOI] | | | 178 |
Nanomaterial mediated optogenetics: opportunities and challenges |
|
| Kai Huang,Qingqing Dou,Xian Jun Loh | | RSC Adv.. 2016; 6(65): 60896 | | [Pubmed] | [DOI] | | | 179 |
Mapping of bionic array electric field focusing in plasmid DNA-based gene electrotransfer |
|
| C J Browne,J L Pinyon,D M Housley,E N Crawford,N H Lovell,M Klugmann,G D Housley | | Gene Therapy. 2016; | | [Pubmed] | [DOI] | | | 180 |
A COMPARISON OF UNILAMELLAR AND MULTILAMELLAR LIPOPLEXES IN TERMS OF TRANSFECTION EFFICIENCY OF HUMAN EMBRYONIC KIDNEY CELLS |
|
| Mohmood Tavallaie | | Journal of Nanotechnology and Materials Science. 2016; 3(2): 1 | | [Pubmed] | [DOI] | | | 181 |
Improvement of vascular function by magnetic nanoparticle-assisted circumferential gene transfer into the native endothelium |
|
| Sarah Vosen,Sarah Rieck,Alexandra Heidsieck,Olga Mykhaylyk,Katrin Zimmermann,Christian Plank,Bernhard Gleich,Alexander Pfeifer,Bernd K. Fleischmann,Daniela Wenzel | | Journal of Controlled Release. 2016; | | [Pubmed] | [DOI] | | | 182 |
Gene transfer to the outflow tract |
|
| Yalong Dang,Ralitsa Loewen,Hardik A. Parikh,Pritha Roy,Nils A. Loewen | | Experimental Eye Research. 2016; | | [Pubmed] | [DOI] | | | 183 |
ORMOPLEXEs for gene therapy: In vitro and in vivo assays |
|
| J.C. Matos,A.R. Soares,I. Domingues,G.A. Monteiro,M.C. Gonçalves | | Materials Science and Engineering: C. 2016; 63: 546 | | [Pubmed] | [DOI] | | | 184 |
Therapeutic and diagnostic applications of extracellular vesicles |
|
| Stephan Stremersch,Stefaan C. De Smedt,Koen Raemdonck | | Journal of Controlled Release. 2016; | | [Pubmed] | [DOI] | | | 185 |
Therapeutic nucleic acids: current clinical status |
|
| Kannan Sridharan,Nithya Jaideep Gogtay | | British Journal of Clinical Pharmacology. 2016; 82(3): 659 | | [Pubmed] | [DOI] | | | 186 |
Improved biocompatibility and efficient labeling of neural stem cells with poly(L-lysine)-coated maghemite nanoparticles |
|
| Igor M Pongrac,Marina Dobrivojevic,Lada Brkic Ahmed,Michal Babic,Miroslav Šlouf,Daniel Horák,Srecko Gajovic | | Beilstein Journal of Nanotechnology. 2016; 7: 926 | | [Pubmed] | [DOI] | | | 187 |
New polymer of lactic-co-glycolic acid-modified polyethylenimine for nucleic acid delivery |
|
| Jian-Ming Lü,Zhengdong Liang,Xiaoxiao Wang,Jianhua Gu,Qizhi Yao,Changyi Chen | | Nanomedicine. 2016; 11(15): 1971 | | [Pubmed] | [DOI] | | | 188 |
Integrated Electrowetting Nanoinjector for Single Cell Transfection |
|
| Elaheh Shekaramiz,Ganeshkumar Varadarajalu,Philip J. Day,H. Kumar Wickramasinghe | | Scientific Reports. 2016; 6: 29051 | | [Pubmed] | [DOI] | | | 189 |
Coordinative Amphiphiles as Tunable siRNA Transporters |
|
| Jin Bum Kim,Yeong Mi Lee,Jooyeon Ryu,Eunji Lee,Won Jong Kim,Gyochang Keum,Eun-Kyoung Bang | | Bioconjugate Chemistry. 2016; | | [Pubmed] | [DOI] | | | 190 |
Gene based therapies for kidney regeneration |
|
| Manoe J. Janssen,Fanny O. Arcolino,Perry Schoor,Robbert Jan Kok,Enrico Mastrobattista | | European Journal of Pharmacology. 2016; | | [Pubmed] | [DOI] | | | 191 |
Micro and nanotechnologies in heart valve tissue engineering |
|
| Anwarul Hasan,John Saliba,Hassan Pezeshgi Modarres,Ahmed Bakhaty,Amir Nasajpour,Mohammad R.K. Mofrad,Amir Sanati-Nezhad | | Biomaterials. 2016; 103: 278 | | [Pubmed] | [DOI] | | | 192 |
Virus-inspired nucleic acid delivery system: Linking virus and viral mimicry |
|
| Rong Ni,Junli Zhou,Naushad Hossain,Ying Chau | | Advanced Drug Delivery Reviews. 2016; | | [Pubmed] | [DOI] | | | 193 |
High-throughput screening of clinically approved drugs that prime polyethylenimine transfection reveals modulation of mitochondria dysfunction response improves gene transfer efficiencies |
|
| Albert Nguyen,Jared Beyersdorf,Jean-Jack Riethoven,Angela K. Pannier | | Bioengineering & Translational Medicine. 2016; | | [Pubmed] | [DOI] | | | 194 |
A novel non-viral gene vector for hepatocyte-targeting and in situ monitoring of DNA delivery in single cells |
|
| Yong-Guang Gao,Quan Tang,You-Di Shi,Ying Zhang,Ruibing Wang,Zhong-Lin Lu | | RSC Adv.. 2016; 6(55): 50053 | | [Pubmed] | [DOI] | | | 195 |
MicroRNAs Regulate Cytokine Responses in Gingival Epithelial Cells |
|
| Steven C. Y. Chen,Christos Constantinides,Moritz Kebschull,Panos N. Papapanou,B. A. McCormick | | Infection and Immunity. 2016; 84(12): 3282 | | [Pubmed] | [DOI] | | | 196 |
An Electrostatically Self-Assembled Ternary Nanocomplex as a Non-Viral Vector for the Delivery of Plasmid DNA into Human Adipose-Derived Stem Cells |
|
| Sun-Hee Cho,Young-Woock Noh,Mi Cho,Yong Lim | | Molecules. 2016; 21(5): 572 | | [Pubmed] | [DOI] | | | 197 |
Delivery of drugs and macromolecules to the mitochondria for cancer therapy |
|
| Phong Lu,Benjamin J. Bruno,Malena Rabenau,Carol S. Lim | | Journal of Controlled Release. 2015; | | [Pubmed] | [DOI] | | | 198 |
A naphthalimide-based [12]aneN3compound as an effective and real-time fluorescence tracking non-viral gene vector |
|
| Yong-Guang Gao,You-Di Shi,Ying Zhang,Jing Hu,Zhong-Lin Lu,Lan He | | Chem. Commun.. 2015; 51(93): 16695 | | [Pubmed] | [DOI] | | | 199 |
Iron Oxide Nanoparticles Coated with a Phosphorothioate Oligonucleotide and a Cationic Peptide: Exploring Four Different Ways of Surface Functionalization |
|
| Frédéric Geinguenaud,Claire Banissi,Antoine Carpentier,Laurence Motte | | Nanomaterials. 2015; 5(4): 1588 | | [Pubmed] | [DOI] | | | 200 |
Nanotechnology Approaches for the Delivery of Exogenous siRNA for HIV Therapy |
|
| Simeon K. Adesina,Emmanuel O. Akala | | Molecular Pharmaceutics. 2015; | | [Pubmed] | [DOI] | | | 201 |
Poly(ethylenimine) conjugated bioreducible dendrimer for efficient gene delivery |
|
| Kihoon Nam,Simhyun Jung,Joung-Pyo Nam,Sung Wan Kim | | Journal of Controlled Release. 2015; 220: 447 | | [Pubmed] | [DOI] | | | 202 |
Highly luminescent and cytocompatible cationic Ag2S NIR-emitting quantum dots for optical imaging and gene transfection |
|
| Fatma Demir Duman,Ibrahim Hocaoglu,Deniz Gulfem Ozturk,Devrim Gozuacik,Alper Kiraz,Havva Yagci Acar | | Nanoscale. 2015; 7(26): 11352 | | [Pubmed] | [DOI] | | | 203 |
Synthesis of bifunctional molecules containing [12]aneN3and coumarin moieties as effective DNA condensation agents and new non-viral gene vectors |
|
| Pan Yue,Ying Zhang,Zhi-Fo Guo,Ao-Cheng Cao,Zhong-Lin Lu,Yong-Gong Zhai | | Org. Biomol. Chem.. 2015; 13(15): 4494 | | [Pubmed] | [DOI] | | | 204 |
Generation of stable cell line by using chitosan as gene delivery system |
|
| Emine Salva,Suna Özbas Turan,Ceyda Ekentok,Jülide Akbuga | | Cytotechnology. 2015; | | [Pubmed] | [DOI] | | | 205 |
Anticancer effect of gene/peptide co-delivery system using transferrin-grafted LMWSC |
|
| Gyeong-Won Jeong,Seong-Cheol Park,Changyong Choi,Joung-Pyo Nam,Tae-Hun Kim,Soo-Kyung Choi,Jun-Kyu Park,Jae-Woon Nah | | International Journal of Pharmaceutics. 2015; 488(1-2): 165 | | [Pubmed] | [DOI] | | | 206 |
Nanotechnology for mesenchymal stem cell therapies |
|
| Bruna Corradetti,Mauro Ferrari | | Journal of Controlled Release. 2015; | | [Pubmed] | [DOI] | | | 207 |
Enhancement of nucleic acid delivery to hard-to-transfect human colorectal cancer cells by magnetofection at laminin coated substrates and promotion of the endosomal/lysosomal escape |
|
| María Belén Cerda,Milena Batalla,Martina Anton,Eduardo Cafferata,Osvaldo Podhajcer,Christian Plank,Olga Mykhaylyk,Lucia Policastro | | RSC Adv.. 2015; 5(72): 58345 | | [Pubmed] | [DOI] | | | 208 |
Gene therapy for ocular diseases meditated by ultrasound and microbubbles (Review) |
|
| Caifeng Wan,Fenghua Li,Hongli Li | | Molecular Medicine Reports. 2015; | | [Pubmed] | [DOI] | | | 209 |
Using Stem Cells to Model Diseases of the Outer Retina |
|
| Camille Yvon,Conor M. Ramsden,Amelia Lane,Michael B. Powner,Lyndon da Cruz,Peter J. Coffey,Amanda-Jayne F. Carr | | Computational and Structural Biotechnology Journal. 2015; 13: 382 | | [Pubmed] | [DOI] | | | 210 |
Polyamidoamine (PAMAM) dendrimers modified with short oligopeptides for early endosomal escape and enhanced gene delivery |
|
| Le Thi Thuy,Sudipta Mallick,Joon Sig Choi | | International Journal of Pharmaceutics. 2015; 492(1-2): 233 | | [Pubmed] | [DOI] | | | 211 |
Stimuli-responsive liposomes for the delivery of nucleic acid therapeutics |
|
| Fatemeh Movahedi,Rebecca G. Hu,David L. Becker,Chenjie Xu | | Nanomedicine: Nanotechnology, Biology and Medicine. 2015; 11(6): 1575 | | [Pubmed] | [DOI] | | | 212 |
Neprilysin gene transfer: A promising therapeutic approach for Alzheimerćs disease |
|
| Yuanli Li,Junqing Wang,Shenghao Zhang,Zhaohui Liu | | Journal of Neuroscience Research. 2015; 93(9): 1325 | | [Pubmed] | [DOI] | | | 213 |
Human umbilical cord blood derived mesenchymal stem cells were differentiated into pancreatic endocrine cell by Pdx-1 electrotransfer |
|
| Phuoc Thi-My Nguyen,Anh Thai-Quynh Nguyen,Nhung Thi Nguyen,Nguyet Thi-Minh Nguyen,Thu Thi Duong,Nhung Hai Truong,Ngoc Kim Phan | | Biomedical Research and Therapy. 2014; 1(2) | | [Pubmed] | [DOI] | | | 214 |
A dual gold nanoparticle system for mesenchymal stem cell tracking |
|
| L. M. Ricles,S. Y. Nam,E. A. Trevińo,S. Y. Emelianov,L. J. Suggs | | J. Mater. Chem. B. 2014; 2(46): 8220 | | [Pubmed] | [DOI] | | | 215 |
Advances in Lipid-Based Platforms for RNAi Therapeutics |
|
| Sara Falsini,Laura Ciani,Sandra Ristori,Angelo Fortunato,Annarosa Arcangeli | | Journal of Medicinal Chemistry. 2014; 57(4): 1138 | | [Pubmed] | [DOI] | | | 216 |
Injection parameters and virus dependent choice of promoters to improve neuron targeting in the nonhuman primate brain |
|
| W Lerchner,B Corgiat,V Der Minassian,R C Saunders,B J Richmond | | Gene Therapy. 2014; | | [Pubmed] | [DOI] | | | 217 |
Effective Gene Delivery into Human Stem Cells with a Cell-Targeting Peptide-Modified Bioreducible Polymer |
|
| Jagadish Beloor,Suresh Ramakrishna,Kihoon Nam,Chang Seon Choi,Jongkil Kim,Sung Hwa Kim,Hyong Jin Cho,HeungSoo Shin,Hyongbum Kim,Sung Wan Kim,Sang-Kyung Lee,Priti Kumar | | Small. 2014; : n/a | | [Pubmed] | [DOI] | | | 218 |
Gene Therapy for Inherited Muscle Diseases |
|
| Robynne Braun,Zejing Wang,David L. Mack,Martin K. Childers | | American Journal of Physical Medicine & Rehabilitation. 2014; 93: S97 | | [Pubmed] | [DOI] | | | 219 |
Gene therapy in pancreatic cancer |
|
| Si-Xue Liu | | World Journal of Gastroenterology. 2014; 20(37): 13343 | | [Pubmed] | [DOI] | | | 220 |
Ultrasound-mediated transgene expression in endogenous stem cells recruited to bone injury sites |
|
| Galina Shapiro,Ilan Kallai,Dmitriy Sheyn,Wafa Tawackoli,Young Do Koh,Hyun Bae,Tamar Trietel,Riki Goldbart,Joseph Kost,Zulma Gazit,Dan Gazit,Gadi Pelled | | Polymers for Advanced Technologies. 2014; : n/a | | [Pubmed] | [DOI] | | | 221 |
Bacteriophages as vehicles for gene delivery into mammalian cells: prospects and problems |
|
| Babak Bakhshinejad,Majid Sadeghizadeh | | Expert Opinion on Drug Delivery. 2014; : 1 | | [Pubmed] | [DOI] | | | 222 |
Polymeric Nanoparticle System to Target activated microglia/macrophages in Spinal Cord Injury |
|
| Simonetta Papa,Raffaele Ferrari,Massimiliano De Paola,Filippo Rossi,Alessandro Mariani,Ilaria Caron,Eliana Sammali,Marco Peviani,Valentina Dell’Oro,Claudio Colombo,Massimo Morbidelli,Gianluigi Forloni,Giuseppe Perale,Davide Moscatelli,Pietro Veglianese | | Journal of Controlled Release. 2013; | | [Pubmed] | [DOI] | | | 223 |
Gene therapy for cardiovascular disease mediated by ultrasound and microbubbles |
|
| Zhi-Yi Chen,Yan Lin,Feng Yang,Lan Jiang,Shu ping Ge | | Cardiovascular Ultrasound. 2013; 11(1): 11 | | [Pubmed] | [DOI] | |
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