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. 2005 Apr;11(4):423-8.
doi: 10.1038/nm1207. Epub 2005 Mar 13.

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

Cédric Raoul  1 , Toufik Abbas-Terki, Jean-Charles Bensadoun, Sandrine Guillot, Georg Haase, Jolanta Szulc, Christopher E Henderson, Patrick Aebischer
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Free article

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

Cédric Raoul et al. Nat Med. 2005 Apr.
Free article

Abstract

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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