Article
Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model.
Nature medicine - 1 Apr 2005
Ralph G Scott, Radcliffe Pippa A, Day Denise M, Carthy Janine M, Leroux Marie A, Lee Debbie C P, Wong Liang-Fong, Bilsland Lynsey G, Greensmith Linda, Kingsman Susan M, Mitrophanous Kyriacos A, Mazarakis Nicholas D, Azzouz Mimoun
Abstract excerpt
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease resulting in the selective death of motor neurons in the brain and spinal cord. Some familial cases of ALS are caused by dominant mutations in the gene encoding superoxide dismutase (SOD1). The emergence of interfering RNA (RNAi) for specific gene silencing could be therapeutically beneficial for the treatment of such dominantly inherited...
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