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

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

Neurofibromatosis type 1 (NF1) is one of the most common genetic disorders caused by heterozygous germline NF1 mutations. NF1 affects many systems, including the skeletal system. To date, no curative therapies are available for skeletal manifestations such as scoliosis and tibial dysplasia, mainly d...

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Bibliographic Details
Main Authors: Annabelle Darle, Thibault Mahiet, Déborah Aubin, Manon Doyen, Lina El Kassar, Béatrice Parfait, Gilles Lemaitre, Christine Baldeschi, Jennifer Allouche, Nathalie Holic
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-02-01
Series:Frontiers in Cell and Developmental Biology
Subjects:
neurofibromatosis type I
gene editing
CRISPR/Cas9
human-induced pluripotent stem cells
osteogenic differentiation
disease modeling
Online Access:https://www.frontiersin.org/articles/10.3389/fcell.2024.1359561/full

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https://www.frontiersin.org/articles/10.3389/fcell.2024.1359561/full

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