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Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

The most common dominantly inherited ataxia, spinocerebellar ataxia type 3 (SCA3), is an incurable neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene that encodes an abnormally long polyglutamine tract in the disease protein, ATXN3. Mice lacking ATXN3 are phenotypically no...

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Detalles Bibliográficos
Autores principales:	Moore, Lauren R., Rajpal, Gautam, Dillingham, Ian T., Qutob, Maya, Blumenstein, Kate G., Gattis, Danielle, Hung, Gene, Kordasiewicz, Holly B., Paulson, Henry L., McLoughlin, Hayley S.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5415970/ https://www.ncbi.nlm.nih.gov/pubmed/28624196 http://dx.doi.org/10.1016/j.omtn.2017.04.005

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