Article
Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2013
Rodríguez-Lebrón Edgardo, Costa Maria do Carmo, Costa Maria doCarmo, Luna-Cancalon Katiuska, Peron Therese M, Fischer Svetlana, Boudreau Ryan L, Davidson Beverly L, Paulson Henry L
Abstract excerpt
Spinocerebellar ataxia type 3 (SCA3) is a neurodegenerative disease caused by a polyglutamine expansion in the deubiquitinating enzyme, Ataxin-3. Currently, there are no effective treatments for this fatal disorder but studies support the hypothesis that reducing mutant Ataxin-3 protein levels might reverse or halt the progression of disease in SCA3. Here, we sought to modulate ATXN3 expression in vivo using RNA...
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