Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide

Published 2019 View Full Article

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Title
Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide
Authors
Keywords
antisense oligonucleotide, SCA1, SCA3, polyglutamine disorders, exon skip, CAG repeat
Journal
Molecular Therapy-Nucleic Acids
Volume 17, Issue -, Pages 601-614
Publisher
Elsevier BV
Online
2019-07-20
DOI
10.1016/j.omtn.2019.07.004

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