CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies.
CRISPR therapy
CRISPR/Cas9
Duchenne muscular dystrophy
animal models
mice models
Journal
WIREs mechanisms of disease
ISSN: 2692-9368
Titre abrégé: WIREs Mech Dis
Pays: United States
ID NLM: 9918227353306676
Informations de publication
Date de publication:
01 2023
01 2023
Historique:
revised:
28
04
2022
received:
02
02
2022
accepted:
30
06
2022
pubmed:
1
8
2022
medline:
17
1
2023
entrez:
31
7
2022
Statut:
ppublish
Résumé
CRISPR gene-editing technology creates precise and permanent modifications to DNA. It has significantly advanced our ability to generate animal disease models for use in biomedical research and also has potential to revolutionize the treatment of genetic disorders. Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disease that could potentially benefit from the development of CRISPR therapy. It is commonly associated with mutations that disrupt the reading frame of the DMD gene that encodes dystrophin, an essential scaffolding protein that stabilizes striated muscles and protects them from contractile-induced damage. CRISPR enables the rapid generation of various animal models harboring mutations that closely simulates the wide variety of mutations observed in DMD patients. These models provide a platform for the testing of sequence-specific interventions like CRISPR therapy that aim to reframe or skip DMD mutations to restore functional dystrophin expression. This article is categorized under: Congenital Diseases > Genetics/Genomics/Epigenetics.
Identifiants
pubmed: 35909075
doi: 10.1002/wsbm.1580
pmc: PMC10078488
doi:
Substances chimiques
Dystrophin
0
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e1580Informations de copyright
© 2022 The Authors. WIREs Mechanisms of Disease published by Wiley Periodicals LLC.
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