Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation.
CRISPR-Cas9
LGMD
MT: RNA/DNA Editing
gene editing
mRNA
stem cell therapy
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
14 Mar 2023
14 Mar 2023
Historique:
received:
17
08
2022
accepted:
01
02
2023
entrez:
3
3
2023
pubmed:
4
3
2023
medline:
4
3
2023
Statut:
epublish
Résumé
With thousands of patients worldwide,
Identifiants
pubmed: 36865086
doi: 10.1016/j.omtn.2023.02.005
pii: S2162-2531(23)00025-2
pmc: PMC9972404
doi:
Types de publication
Journal Article
Langues
eng
Pagination
494-511Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
The authors declare no competing interests.
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