Generation of heterozygous and homozygous NF1 lines from human-induced pluripotent stem cells using CRISPR/Cas9 to investigate bone defects associated with neurofibromatosis type 1.

CRISPR/Cas9 disease modeling gene editing human-induced pluripotent stem cells neurofibromatosis type I osteogenic differentiation

Journal

Frontiers in cell and developmental biology
ISSN: 2296-634X
Titre abrégé: Front Cell Dev Biol
Pays: Switzerland
ID NLM: 101630250

Informations de publication

Date de publication:
2024
Historique:
received: 21 12 2023
accepted: 07 02 2024
medline: 14 3 2024
pubmed: 14 3 2024
entrez: 14 3 2024
Statut: epublish

Résumé

Neurofibromatosis type 1 (NF1) is one of the most common genetic disorders caused by heterozygous germline

Identifiants

pubmed: 38481529
doi: 10.3389/fcell.2024.1359561
pii: 1359561
pmc: PMC10935092
doi:

Types de publication

Journal Article

Langues

eng

Pagination

1359561

Informations de copyright

Copyright © 2024 Darle, Mahiet, Aubin, Doyen, El Kassar, Parfait, Lemaitre, Baldeschi, Allouche and Holic.

Déclaration de conflit d'intérêts

Author DA was employed by company Phenocell SAS. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Auteurs

Annabelle Darle (A)

Centre d'Etude des Cellules Souches, Corbeil-Essonnes, France.

Thibault Mahiet (T)

Centre d'Etude des Cellules Souches, Corbeil-Essonnes, France.

Déborah Aubin (D)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.
Phenocell SAS, Grasse, France.

Manon Doyen (M)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.

Lina El Kassar (L)

Centre d'Etude des Cellules Souches, Corbeil-Essonnes, France.

Béatrice Parfait (B)

Equipe "Génomique et Epigénétique des Tumeurs Rares", UMR INSERM 1016 & Université Paris Cité, Institut Cochin, Paris, France.
GHU AP-HP Centre-Université Paris Cité, Fédération de Médecine Génomique, Service de Médecine Génomique des Maladies de Système et d'Organe, Hôpital Cochin, Paris, France.

Gilles Lemaitre (G)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.

Christine Baldeschi (C)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.

Jennifer Allouche (J)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.

Nathalie Holic (N)

Université Paris-Saclay, Université d'Evry, Corbeil-Essonnes, France.
INSERM U861, I-Stem, Association Française contre les Myopathies (AFM), Institute for Stem Cell Therapy and Exploration of Monogenic Diseases, Corbeil-Essonnes, France.

Classifications MeSH