Molecular medicine for the brain: silencing of disease genes with RNA interference

被引：89

作者：

Davidson, BL ^{[1
]}

Paulson, HL

机构：

[1] Univ Iowa, Dept Internal Med, Iowa City, IA 52242 USA

[2] Univ Iowa, Dept Neurol, Iowa City, IA USA

[3] Univ Iowa, Dept Physiol & Biophys, Iowa City, IA USA

来源：

LANCET NEUROLOGY | 2004年 / 3卷 / 03期

基金：

美国国家卫生研究院;

关键词：

D O I：

10.1016/S1474-4422(04)00678-7

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

The recent discovery of RNA interference (RNAi) has revolutionised biological research and now holds promise as a potential therapy for human diseases. Currently untreatable neurological diseases are especially attractive targets. Scientists have already succeeded in using RNAi to suppress dominant disease genes in vitro; in some cases, this suppression has been allele-specific, silencing the disease-causing allele while maintaining expression of the normal allele. The challenge now is to bring this powerful technology in vivo to animal models to suppress disease genes and correct disease phenotypes. In the confrontation of this challenge, research should benefit from recent advances in viral and non-viral delivery of therapy to the brain.

引用

页码：145 / 149

页数：5

共 33 条

[1] Allele-specific silencing of a pathogenic mutant acetylcholine receptor subunit by RNA interference [J].

Abdelgany, A ;

Wood, M ;

Beeson, D .

HUMAN MOLECULAR GENETICS, 2003, 12 (20) :2637-2644

[2] Convection-enhanced delivery of AAV vector in parkinsonian monkeys;: In vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach [J].

Bankiewicz, KS ;

Eberling, JL ;

Kohutnicka, M ;

Jagust, W ;

Pivirotto, P ;

Bringas, J ;

Cunningham, J ;

Budinger, TF ;

Harvey-White, J .

EXPERIMENTAL NEUROLOGY, 2000, 164 (01) :2-14

[3]

Blomer U, 1997, J VIROL, V71, P6641

[4] Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors [J].

Brooks, AI ;

Stein, CS ;

Hughes, SM ;

Heth, J ;

McCray, PM ;

Sauter, SL ;

Johnston, JC ;

Cory-Slechta, DA ;

Federoff, HJ ;

Davidson, BL .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (09) :6216-6221

[5] 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

[6] Rescue of polyglutamine-mediated cytotoxicity by double-stranded RNA-mediated RNA interference [J].

Caplen, NJ ;

Taylor, JP ;

Statham, VS ;

Tanaka, F ;

Fire, A ;

Morgan, RA .

HUMAN MOLECULAR GENETICS, 2002, 11 (02) :175-184

[7] Genomewide view of gene silencing by small interfering RNAs [J].

Chi, JT ;

Chang, HY ;

Wang, NN ;

Chang, DS ;

Dunphy, N ;

Brown, PO .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (11) :6343-6346

[8] Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system [J].

Davidson, BL ;

Stein, CS ;

Heth, JA ;

Martins, I ;

Kotin, RM ;

Derksen, TA ;

Zabner, J ;

Ghodsi, A ;

Chiorini, JA .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2000, 97 (07) :3428-3432

[9] The specifics of small interfering RNA specificity [J].

Dillin, A .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2003, 100 (11) :6289-6291

[10] Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis [J].

Ding, HL ;

Schwarz, DS ;

Keene, A ;

Affar, EB ;

Fenton, L ;

Xia, XA ;

Shi, Y ;

Zamore, PD ;

Xu, ZS .

AGING CELL, 2003, 2 (04) :209-217

← 1 2 3 4 →