Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy.
DMDmdx rat
Duchenne muscular dystrophy
age
dose study
gene therapy
mini-dystrophin
recombinant adeno-associated virus
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:
14 Sep 2023
14 Sep 2023
Historique:
received:
29
07
2022
accepted:
15
05
2023
medline:
25
9
2023
pubmed:
25
9
2023
entrez:
25
9
2023
Statut:
epublish
Résumé
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by loss-of-function mutations in the
Identifiants
pubmed: 37746247
doi: 10.1016/j.omtm.2023.05.017
pii: S2329-0501(23)00079-7
pmc: PMC10512999
doi:
Types de publication
Journal Article
Langues
eng
Pagination
30-47Informations de copyright
© 2023 The Authors.
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