Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach.
CRISPR interference
CRISPR-Cas9
DMPK
RNA foci
genome editing
myotonic dystrophy
off-target
Journal
Molecular therapy. Methods & clinical development
ISSN: 2329-0501
Titre abrégé: Mol Ther Methods Clin Dev
Pays: United States
ID NLM: 101624857
Informations de publication
Date de publication:
11 Sep 2020
11 Sep 2020
Historique:
received:
12
05
2020
accepted:
20
05
2020
entrez:
9
7
2020
pubmed:
9
7
2020
medline:
9
7
2020
Statut:
epublish
Résumé
Myotonic dystrophy type 1 is the most common type of adult-onset muscular dystrophy. This is an autosomal dominant disorder and caused by the expansion of the CTG repeat in the 3' untranslated region of the dystrophia myotonica protein kinase (
Identifiants
pubmed: 32637445
doi: 10.1016/j.omtm.2020.05.024
pii: S2329-0501(20)30111-X
pmc: PMC7321784
doi:
Types de publication
Journal Article
Langues
eng
Pagination
131-144Informations de copyright
© 2020 The Author(s).
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