Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

• Главные авторы: Lauren R. Moore (Автор), Gautam Rajpal (Автор), Ian T. Dillingham (Автор), Maya Qutob (Автор), Kate G. Blumenstein (Автор), Danielle Gattis (Автор), Gene Hung (Автор), Holly B. Kordasiewicz (Автор), Henry L. Paulson (Автор), Hayley S. McLoughlin (Автор)
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• Опубликовано: Elsevier, 2017-06-01T00:00:00Z.
• Предметы: article
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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...