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- Title
Getting a handle on Huntington's disease: silencing neurodegeneration.
- Authors
La Spada, Albert R.
- Abstract
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.
- Subjects
HUNTINGTON'S chorea treatment; NEURODEGENERATION; GENE silencing; NEUROGENETICS; LABORATORY mice; FRIEDREICH'S ataxia
- Publication
Nature Medicine, 2009, Vol 15, Issue 3, p252
- ISSN
1078-8956
- Publication type
Article
- DOI
10.1038/nm0309-252