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rcharlton

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My Friend in Med school who is focusing her studies on ALS believes that this study is the most important development in ALS research for some time.

The study involved mice subject to a familial form of ALS and the researchers were able to stop the progression of ALS using small interfering RNA.

Now I know this may never have application to humans - and even if it did we are a long way away - still it is very promising.

Here is the synopsis:

J Biol Chem. 2005 Dec 30;280(52):42826-30. Epub 2005 Oct 12. Related Articles, Links

Transgenic small interfering RNA halts amyotrophic lateral sclerosis in a mouse model.

Saito Y, Yokota T, Mitani T, Ito K, Anzai M, Miyagishi M, Taira K, Mizusawa H.

Department of Neurology and Neurological Science, Graduate School, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo 113-8519.

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 small interfering RNA (siRNA), has recently emerged as a powerful gene silencing tool. We made transgenic (Tg) mice with modified siRNA, which had multiple mismatch alternations within the sense strand, to prevent the "shutdown phenomenon" of transgenic siRNA. Consequently, the in vivo knockdown effect of siRNA on SOD1 expression did not diminish over four generations. When we crossed these anti-SOD1 siRNA Tg mice with SOD1G93A Tg mice, a model for ALS, siRNA prevented the development of disease by inhibiting mutant G93A SOD1 production in the central nervous system. Our findings clearly proved the principle that siRNA-mediated gene silencing can stop the development of familial ALS with SOD1 mutation.
 
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