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

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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https://www.hal.inserm.fr/inserm-00484530
Contributor : Roman Tyzio <>
Submitted on : Tuesday, May 18, 2010 - 3:28:32 PM
Last modification on : Tuesday, February 4, 2020 - 10:00:04 AM
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Cédric Raoul, Toufik Abbas-Terki, Jean-Charles Bensadoun, Sandrine Guillot, Georg Haase, et al.. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.. Nature Medicine, Nature Publishing Group, 2005, 11 (4), pp.423-8. ⟨10.1038/nm1207⟩. ⟨inserm-00484530⟩

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