Functional benefit of CRISPR-Cas9-induced allele deletion for

CRISPR-Cas9 MT: RNA/DNA Editing SNP allele deletion calcium release dominant mutation gene therapy myopathy ryanodine receptor

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:
10 Sep 2024
Historique:
received: 08 02 2024
accepted: 14 06 2024
medline: 29 7 2024
pubmed: 29 7 2024
entrez: 29 7 2024
Statut: epublish

Résumé

More than 700 pathogenic or probably pathogenic variations have been identified in the

Identifiants

DOI: 10.1016/j.omtn.2024.102259 PMID: 39071953 PMC: PMC11278293
pubmed: 39071953
doi: 10.1016/j.omtn.2024.102259
pii: S2162-2531(24)00146-X
pmc: PMC11278293
doi:

Types de publication

Journal Article

Langues

eng

Pagination

102259

Informations de copyright

© 2024 The Author(s).

Déclaration de conflit d'intérêts

The authors declare no competing interests.

Auteurs

Mathilde Beaufils (M)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

Margaux Melka (M)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

Julie Brocard (J)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

Clement Benoit (C)

University Grenoble Alpes, TIMC, CNRS UMR5525, 38000 Grenoble, France.

Nagi Debbah (N)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.
University Grenoble Alpes, TIMC, CNRS UMR5525, 38000 Grenoble, France.
University Grenoble Alpes, Departement de Pharmacochimie Moléculaire, CNRS UMR 5063, 38400 Saint-Martin-d'Hères, France.

Kamel Mamchaoui (K)

Sorbonne Université, INSERM, Institut de Myologie, Centre de Recherche en Myologie, 75000 Paris, France.

Norma B Romero (NB)

Neuromuscular Morphology Unit, Institut de Myologie, Pitié-Salpêtrière Hospital, Sorbonne Université, 75000 Paris, France.

Anne Frédérique Dalmas-Laurent (AF)

MH Unit, Lille University Hospital, 59000 Lille, France.

Susana Quijano-Roy (S)

Neuromuscular Unit (NEIDF), Child Neurology and ICU Department, Raymond-Poincaré Hospital, (APHP University Paris-Saclay), 92380 Garches, France.

Julien Fauré (J)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

John Rendu (J)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

Isabelle Marty (I)

University Grenoble Alpes, INSERM, U1216, CHU Grenoble Alpes, Grenoble Institut Neurosciences, 38000 Grenoble, France.

Classifications MeSH