CRISPR/Cas correction of muscular dystrophies.
Duchenne muscular dystrophy
Facioscapulohumeral muscular dystrophy
Genome editing
Limb-girdle muscular dystrophy
Skeletal muscle
sgRNA
Journal
Experimental cell research
ISSN: 1090-2422
Titre abrégé: Exp Cell Res
Pays: United States
ID NLM: 0373226
Informations de publication
Date de publication:
01 11 2021
01 11 2021
Historique:
received:
10
06
2021
revised:
10
09
2021
accepted:
23
09
2021
pubmed:
28
9
2021
medline:
30
11
2021
entrez:
27
9
2021
Statut:
ppublish
Résumé
Muscular dystrophies are a heterogeneous group of monogenic neuromuscular disorders which lead to progressive muscle loss and degeneration of the musculoskeletal system. The genetic causes of muscular dystrophies are well characterized, but no effective treatments have been developed so far. The discovery and application of the CRISPR/Cas system for genome editing offers a new path for disease treatment with the potential to permanently correct genetic mutations. The post-mitotic and multinucleated features of skeletal muscle provide an ideal target for CRISPR/Cas therapeutic genome editing because correction of a subpopulation of nuclei can provide benefit to the whole myofiber. In this review, we provide an overview of the CRISPR/Cas system and its derivatives in genome editing, proposing potential CRISPR/Cas-based therapies to correct diverse muscular dystrophies, and we discuss challenges for translating CRISPR/Cas genome editing to a viable therapy for permanent correction of muscular dystrophies.
Identifiants
pubmed: 34571006
pii: S0014-4827(21)00398-0
doi: 10.1016/j.yexcr.2021.112844
pmc: PMC8530959
mid: NIHMS1745381
pii:
doi:
Substances chimiques
Dystrophin
0
Types de publication
Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
112844Subventions
Organisme : NICHD NIH HHS
ID : P50 HD087351
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR067294
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL130253
Pays : United States
Informations de copyright
Copyright © 2021 Elsevier Inc. All rights reserved.
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