Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

• Main Authors: Lauren R. Moore (Author), Gautam Rajpal (Author), Ian T. Dillingham (Author), Maya Qutob (Author), Kate G. Blumenstein (Author), Danielle Gattis (Author), Gene Hung (Author), Holly B. Kordasiewicz (Author), Henry L. Paulson (Author), Hayley S. McLoughlin (Author)
• Format: Book
• Published: Elsevier, 2017-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...