Developing Gene Silencing for the Study and Treatment of Dystonia
Technical Report,30 Sep 2014,29 Sep 2015
The Children's Hospital of Philadelphia Philadelphia United States
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Dystonia is a debilitating neurological disease with no cure. In dystonia, there are involuntary muscle contractions that cause abnormal twisting postures. DYT1 dystonia is an autosomal dominant disease with onset of dystonia during childhood. The most common early onset inherited dystonia, DYT1 is caused by a common deletion of a single amino acid E in torsinA. There is abundant evidence suggesting that suppressing expression of torsinAE through gene silencing techniques would be beneficial. We have already achieved this goal in cultured cells through RNA interference RNAi and antisense oligonucleotides ASO.