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Article

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

12 Apr 2017

Abstract excerpt

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 normal; hence, disease gene suppression offers a compelling approach to slow the neurodegenerative cascade in SCA3....

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PMID: 28624196
DOI: 10.1016/j.omtn.2017.04.005

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