Disease modeling and gene correction of LGMDR21 iPSCs elucidates the role of POGLUT1 in skeletal muscle maintenance, regeneration, and the satellite cell niche

Disease modeling and gene correction of LGMDR21 iPSCs elucidates the role of POGLUT1 in skeletal muscle maintenance, regeneration, and the satellite cell niche

Autosomal recessive limb-girdle muscular dystrophy 21 (LGMDR21) is caused by pathogenic variants in protein O-glucosyltransferase 1 (POGLUT1), which is responsible for O-glucosylation of specific epidermal growth factor (EGF) repeats found in ∼50 mammalian proteins, including Notch receptors. Previo...

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Bibliographic Details
Main Authors: Jose L. Ortiz-Vitali, Jianbo Wu, Nasa Xu, Annie W. Shieh, Nima Niknejad, Megumi Takeuchi, Carmen Paradas, Chunru Lin, Hamed Jafar-Nejad, Robert S. Haltiwanger, Sidney H. Wang, Radbod Darabi
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
POGLUT1
LGMDR21
iPSCs
gene correction
CRISPR-Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312300210X

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http://www.sciencedirect.com/science/article/pii/S216225312300210X

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