CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing

Published 2017 View Full Article

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Title
CRISPR/Cas9-Induced (CTG⋅CAG) n Repeat Instability in the Myotonic Dystrophy Type 1 Locus: Implications for Therapeutic Genome Editing
Authors
Keywords
myotonic dystrophy, trinucleotide instability, (CTG⋅CAG)n repeat, DM1 myoblasts, CRISPR/Cas9, therapeutic genome editing, NHEJ, dsDNA break repair, somatic cell therapy
Journal
MOLECULAR THERAPY
Volume 25, Issue 1, Pages 24-43
Publisher
Elsevier BV
Online
2017-01-06
DOI
10.1016/j.ymthe.2016.10.014

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