TY - JOUR
T1 - Polymers in gene therapy technology
AU - Hosseinkhani, Hossein
AU - Abedini, Fatemeh
AU - Ou, Keng Liang
AU - Domb, Abraham J.
N1 - Publisher Copyright:
© 2014 John Wiley & Sons, Ltd.
PY - 2015/2/1
Y1 - 2015/2/1
N2 - There are several barriers to gene delivery. One of the biggest challenges is the design of appropriate vectors. Currently, nonviral vectors have received significant attention because of low toxicity, potential for tissue specificity, stability during storage, lack of immunogenicity, and relatively low production cost. Despite the high efficiency of viral vectors, they show limited clinical applications because of potentially fatal adverse effects and because of the likelihood of the immune response shutting down the transgene expression system. Nonviral technologies comprise plasmid-based expression systems harboring a gene that encodes a therapeutic protein along with a synthetic gene delivery system. This review provides a broad perspective on recent improvements in the development of four kinds of nonviral vectors that are based on polymers, peptides, lipids, and DNA and discusses the cytotoxicity associated with gene therapy.
AB - There are several barriers to gene delivery. One of the biggest challenges is the design of appropriate vectors. Currently, nonviral vectors have received significant attention because of low toxicity, potential for tissue specificity, stability during storage, lack of immunogenicity, and relatively low production cost. Despite the high efficiency of viral vectors, they show limited clinical applications because of potentially fatal adverse effects and because of the likelihood of the immune response shutting down the transgene expression system. Nonviral technologies comprise plasmid-based expression systems harboring a gene that encodes a therapeutic protein along with a synthetic gene delivery system. This review provides a broad perspective on recent improvements in the development of four kinds of nonviral vectors that are based on polymers, peptides, lipids, and DNA and discusses the cytotoxicity associated with gene therapy.
KW - Cationic lipids
KW - Cationic polymers
KW - Cell-penetrating peptides
KW - DNA transposon-based vectors
KW - Nonviral gene therapy
KW - Toxicity
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U2 - 10.1002/pat.3432
DO - 10.1002/pat.3432
M3 - Article
AN - SCOPUS:84921321504
SN - 1042-7147
VL - 26
SP - 198
EP - 211
JO - Polymers for Advanced Technologies
JF - Polymers for Advanced Technologies
IS - 2
ER -