Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease

Machado–Joseph disease (MJD) is a dominantly inherited ataxia caused by a polyglutamine-coding expansion in the ATXN3 gene. Suppressing expression of the toxic gene product represents a promising approach to therapy for MJD and other polyglutamine diseases. We performed an extended therapeutic trial...

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Main Authors: do Carmo Costa, Maria, Luna-Cancalon, Katiuska, Fischer, Svetlana, Ashraf, Naila S, Ouyang, Michelle, Dharia, Rahil M, Martin-Fishman, Lucas, Yang, Yemen, Shakkottai, Vikram G, Davidson, Beverly L, Rodríguez-Lebrón, Edgardo, Paulson, Henry L
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2013
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Original Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808129/
https://ncbi.nlm.nih.gov/pubmed/23765441
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.144
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