Article
Transgenic small interfering RNA halts amyotrophic lateral sclerosis in a mouse model.
The Journal of biological chemistry - 30 Dec 2005
Saito Yuki, Yokota Takanori, Mitani Tasuku, Ito Kaoru, Anzai Masayuki, Miyagishi Makoto, Taira Kazunari, Mizusawa Hidehiro
Abstract excerpt
Many autosomal dominant diseases such as familial amyotrophic lateral sclerosis (ALS) with copper/zinc superoxide dismutase (SOD1) mutation may be induced by missense point mutations that result in the production of proteins with toxic properties. Reduction in the encoding of proteins from such mutated genes can therefore be expected to improve the disease phenotype. The duplex of 21-nucleotide RNA, known as...
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