Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

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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Bibliographic Details
Main Authors: Lauren R. Moore, Gautam Rajpal, Ian T. Dillingham, Maya Qutob, Kate G. Blumenstein, Danielle Gattis, Gene Hung, Holly B. Kordasiewicz, Henry L. Paulson, Hayley S. McLoughlin
Format: Article
Language:English
Published: Elsevier 2017-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligonucleotide
ASO
ATXN3
spinocerebellar ataxia type 3
SCA3
polyglutamine disease
Machado-Joseph disease
MJD
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301555

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http://www.sciencedirect.com/science/article/pii/S2162253117301555

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