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

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 expres...

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Published inNature Medicine Vol. 11; no. 43; pp. 423 - 428
Main Authors Aebischer, Patrick, Raoul, Cédric, Abbas-Terki, Toufik, Bensadoun, Jean-Charles, Guillot, Sandrine, Haase, Georg, Szulc, Jolanta, Henderson, Christopher E
Format Journal Article Magazine Article
LanguageEnglish
Published United States Nature Publishing Group 01.04.2005
Subjects
Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis - genetics
Animals
Cellular Biology
Disease
Disease Models, Animal
Disease Progression
Fibroblasts
Genetic Vectors
Humans
Infections
Injection
Lentivirus
Life Sciences
Mice
Mice, Transgenic
Molecular Sequence Data
Mutation
RNA Interference
RNA, Small Interfering
Spinal cord
Superoxide Dismutase
Superoxide Dismutase - genetics
Toxicity
Transgenic animals
France
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Summary: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.
Bibliography:ObjectType-Article-2
SourceType-Scholarly Journals-1
ObjectType-Feature-1
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ISSN:1078-8956
1546-170X
1744-7933
DOI:10.1038/nm1207