Lentiviral Vector-Mediated RNA Silencing in the Central Nervous System

被引：27

作者：

Hutson, Thomas H. ^{[1
]}

Foster, Edmund ^{[2
]}

Moon, Lawrence D. F. ^{[1
]}

Yanez-Munoz, Rafael J. ^{[3
]}

机构：

[1] Kings Coll London, Neurorestorat Grp, Wolfson Ctr Age Related Dis, London SE1 1UL, England

[2] Univ Zurich, Inst Pharmacol & Toxicol, CH-8057 Zurich, Switzerland

[3] Royal Holloway Univ London, Sch Biol Sci, Ctr Biomed Sci, Egham TW20 OEX, Surrey, England

来源：

HUMAN GENE THERAPY METHODS | 2014年 / 25卷 / 01期

基金：

英国生物技术与生命科学研究理事会;

关键词：

IMMUNODEFICIENCY-VIRUS TYPE-1; SMALL INTERFERING RNAS; SHORT HAIRPIN RNAS; DOUBLE-STRANDED-RNA; NF-KAPPA-B; MACHADO-JOSEPH-DISEASE; INNATE IMMUNE-RESPONSE; DORSAL-ROOT GANGLION; EXPANDED CAG REPEATS; VIVO GENE DELIVERY;

D O I：

10.1089/hgtb.2013.016

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

RNA silencing is an established method for investigating gene function and has attracted particular interest because of the potential for generating RNA-based therapeutics. Using lentiviral vectors as an efficient delivery system that offers stable, long-term expression in postmitotic cells further enhances the applicability of an RNA-based gene therapy for the CNS. In this review we provide an overview of both lentiviral vectors and RNA silencing along with design considerations for generating lentiviral vectors capable of RNA silencing. We go on to describe the current preclinical data regarding lentiviral vector-mediated RNA silencing for CNS disorders and discuss the concerns of side effects associated with lentiviral vectors and small interfering RNAs and how these might be mitigated.

引用

页码：14 / 32

页数：19

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