Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS - PubMed (original) (raw)
doi: 10.1038/nm1207. Epub 2005 Mar 13.
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- PMID: 15768028
- DOI: 10.1038/nm1207
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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.
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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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