Delivering the code: polyplex carriers for deoxyribonucleic acid and ribonucleic acid interference therapies.
Nucleic acid-based therapies offer great potential for treatment of a variety of diseases including cancer by modulating protein expression with DNA or small interfering RNA. However, realization of their full therapeutic potential is currently limited due to an inability to reach the target site in an active form. Identification of delivery barriers such as stability in circulation, resistance to degradation and entrapment in subcellular vesicles has led to development of sophisticated multifunctional synthetic polymers for forming ionic complexes with nucleic acids and also providing performance-enhancing features. The most promising designs comprise features to help increase stability in circulation and also contain functionality to aid in endosome escape of nucleic acid cargo after cellular internalization.
Christie
R James
RJ
Department of Materials Engineering, Graduate School of Medicine, University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo 113-8656, Japan.
Nishiyama
N
N
Kataoka
K
K
D000818:Animals; D057005:Bioengineering; D003432:Cross-Linking Reagents; D004247:DNA / Q000235:genetics* / Q000627:therapeutic use; D004220:Disulfides; D004337:Drug Carriers; D016503:Drug Delivery Systems; D005786:Gene Expression Regulation; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D051379:Mice; D036103:Nanotechnology / Q000379:methods; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D034741:RNA, Small Interfering / Q000235:genetics* / Q000627:therapeutic use
D003432:Cross-Linking Reagents; D004220:Disulfides; D004337:Drug Carriers; D034741:RNA, Small Interfering; D004247:DNA