Article
Splice isoform-specific suppression of the Cav2.1 variant underlying spinocerebellar ataxia type 6.
Neurobiology of disease - 1 Sept 2011
Tsou Wei-Ling, Soong Bing-Wen, Paulson Henry L, Rodríguez-Lebrón Edgardo
Abstract excerpt
Spinocerebellar ataxia type 6 (SCA6) is an inherited neurodegenerative disease caused by a polyglutamine (polyQ) expansion in the Ca(V)2.1 voltage-gated calcium channel subunit (CACNA1A). There is currently no treatment for this debilitating disorder and thus a pressing need to develop preventative therapies. RNA interference (RNAi) has proven effective at halting disease progression in several models of...
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