Getting a handle on Huntington's disease: silencing neurodegeneration

La Spada, Albert R.
March 2009
Nature Medicine;Mar2009, Vol. 15 Issue 3, p252
Academic Journal
The article focuses on the studies regarding the Huntington's disease, an inherited neurological disorder caused by a dominant gain-of-function gene mutation. It notes that researchers used a mouse model to test whether termination of expression of the mutant gene product would stop disease progression of Huntington's and in spinocerebellar ataxia type 1 (SCA1). Furthermore, a substantial degree of gene silencing in adults without side effects was proved in the RNAi knockdown studies.


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