Myogenesis defects in a patient-derived iPSC model of hereditary GNE myopathy

Myogenesis defects in a patient-derived iPSC model of hereditary GNE myopathy

Abstract Hereditary muscle diseases are disabling disorders lacking effective treatments. UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase (GNE) myopathy (GNEM) is an autosomal recessive distal myopathy with rimmed vacuoles typically manifesting in late adolescence/early adulthood. GNE...

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Bibliographic Details
Main Authors: Rebecca E. Schmitt, Douglas Y. Smith, Dong Seong Cho, Lindsey A. Kirkeby, Zachary T. Resch, Teerin Liewluck, Zhiyv Niu, Margherita Milone, Jason D. Doles
Format: Article
Language:English
Published: Nature Portfolio 2022-09-01
Series:npj Regenerative Medicine
Online Access:https://doi.org/10.1038/s41536-022-00238-3

Internet

https://doi.org/10.1038/s41536-022-00238-3

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