Article
Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression.
The Journal of biological chemistry - 6 Jun 2008
Wang Hongyan, Ghosh Animesh, Baigude Huricha, Yang Chao-Shun, Qiu Linghua, Xia Xugang, Zhou Hongxia, Rana Tariq M, Xu Zuoshang
Abstract excerpt
Inherited neurodegenerative diseases, such as Huntington disease and subset of Alzheimer disease, Parkinson disease, and amyotrophic lateral sclerosis, are caused by the mutant genes that have gained undefined properties that harm cells in the nervous system, causing neurodegeneration and clinical phenotypes. Lowering the mutant gene expression is predicted to slow the disease progression and produce clinical...
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