Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice

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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Rodríguez-Lebrón, Edgardo, Costa, Maria doCarmo, Luna-Cancalon, Katiuska, Peron, Therese M, Fischer, Svetlana, Boudreau, Ryan L, Davidson, Beverly L, Paulson, Henry L
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Nature Publishing Group 2013
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808130/
https://ncbi.nlm.nih.gov/pubmed/23820820
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.152
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