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

C Raoul, T Abbas-Terki, JC Bensadoun, S Guillot… - Nature medicine, 2005 - nature.com
C Raoul, T Abbas-Terki, JC Bensadoun, S Guillot, G Haase, J Szulc, CE Henderson…
Nature medicine, 2005nature.com
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 …
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 SOD1G93A 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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