The therapeutic potential of RNA interference

被引：149

作者：

Uprichard, SL ^{[1
]}

机构：

[1] Scripps Res Inst, Dept Mol & Expt Med, La Jolla, CA 92037 USA

来源：

FEBS LETTERS | 2005年 / 579卷 / 26期

关键词：

gene suppression; drug development; delivery; safety; RNA interference;

D O I：

10.1016/j.febslet.2005.08.004

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

In recent years, we have witnessed the discovery of a new mechanism of gene regulation called RNA interference (RNAi), which has revitalized interest in the development of nocleic acid-based technologies for therapeutic gene suppression. This review focuses on the potential therapeutic use of RNAi, discussing the theoretical advantages of RNAi-based therapeutics over previous technologies as well as the challenges involved in developing RNAi for clinical use. Also reviewed, are the in vivo proof-of principle experiments that provide the preclinical justification for the continued development of RNAi-based therapeutics. (c) 2005 Federation of European Biochemical Societies. Published by Elsevier B.V. All rights reserved.

引用

页码：5996 / 6007

页数：12

共 173 条

[11]

Berkhout B, 2004, CURR OPIN MOL THER, V6, P141

[12] Comparison of antisense oligonucleotides and siRNAs in cell culture and in vivo [J].

Bertrand, JR ;

Pottier, M ;

Vekris, A ;

Opolon, P ;

Maksimenko, A ;

Malvy, C .

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, 2002, 296 (04) :1000-1004

[13] Inhibition of respiratory viruses by nasally administered siRNA [J].

Bitko, V ;

Musiyenko, A ;

Shulyayeva, O ;

Barik, S .

NATURE MEDICINE, 2005, 11 (01) :50-55

[14] Phenotypic silencing of cytoplasmic genes using sequence-specific double-stranded short interfering RNA and its application in the reverse genetics of wild type negative-strand RNA viruses [J].

Bitko V. ;

Barik S. .

BMC Microbiology, 1 (1) :1-11

[15] Human immunodeficiency virus type 1 escape from RNA interference [J].

Boden, D ;

Pusch, O ;

Lee, F ;

Tucker, L ;

Ramratnam, B .

JOURNAL OF VIROLOGY, 2003, 77 (21) :11531-11535

[16] RNA interference in mammalian cells by chemically-modified RNA [J].

Braasch, DA ;

Jensen, S ;

Liu, YH ;

Kaur, K ;

Arar, K ;

White, MA ;

Corey, DR .

BIOCHEMISTRY, 2003, 42 (26) :7967-7975

[17] Stable suppression of tumorigenicity by virus-mediated RNA interference [J].

Brummelkamp, TR ;

Bernards, R ;

Agami, R .

CANCER CELL, 2002, 2 (03) :243-247

[18] A system for stable expression of short interfering RNAs in mammalian cells [J].

Brummelkamp, TR ;

Bernards, R ;

Agami, R .

SCIENCE, 2002, 296 (5567) :550-553

[19] RNA interference: from model organisms towards therapy for neural and neuromuscular disorders [J].

Buckingham, SD ;

Esmaeili, B ;

Wood, M ;

Sattelle, DB .

HUMAN MOLECULAR GENETICS, 2004, 13 :R275-R288

[20] SCA1 TRANSGENIC MICE - A MODEL FOR NEURODEGENERATION CAUSED BY AN EXPANDED CAG TRINUCLEOTIDE REPEAT [J].

BURRIGHT, EN ;

CLARK, HB ;

SERVADIO, A ;

MATILLA, T ;

FEDDERSEN, RM ;

YUNIS, WS ;

DUVICK, LA ;

ZOGHBI, HY ;

ORR, HT .

CELL, 1995, 82 (06) :937-948

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