Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS

被引：343

作者：

Raoul, C

Abbas-Terki, T

Bensadoun, JC

Guillot, S

Haase, G

Szulc, J

Henderson, CE

Aebischer, P ^{[1
]}

机构：

[1] Ecole Polytech Fed Lausanne, Integrat Biosci Inst, CH-1015 Lausanne, Switzerland

[2] INSERM, U29, Avenir Team, INMED, F-13273 Marseille, France

[3] Dev Biol Inst Marseille, INSERM, UMR 623, F-13288 Marseille, France

来源：

NATURE MEDICINE | 2005年 / 11卷 / 04期

关键词：

D O I：

10.1038/nm1207

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：

Mutations in Cu/Zn superoxide dismutase ( encoded by SOD1), one of the causes of familial amyotrophic lateral sclerosis (ALS), lead to progressive death of motoneurons through a gain-of-function mechanism. RNA interference (RNAi) mediated by viral vectors allows for long-term reduction in gene expression and represents an attractive therapeutic approach for genetic diseases characterized by acquired toxic properties. We report that in SOD1(G93A) transgenic mice, a model for familial ALS, intraspinal injection of a lentiviral vector that produces RNAi-mediated silencing of SOD1 substantially retards both the onset and the progression rate of the disease.

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页码：423 / 428

页数：6

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