A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy‐Affected Skeletal Muscles | Zendy

Caron Leslie | Zendy; Kher Devaki | Zendy; Lee Kian Leong | Zendy; McKernan Robert | Zendy; Dumevska Biljana | Zendy; Hidalgo Alejandro | Zendy; Li Jia | Zendy; Yang Henry | Zendy; Main Heather | Zendy; Ferri Giulia | Zendy; Petek Lisa M. | Zendy; Poellinger Lorenz | Zendy; Miller Daniel G. | Zendy; Gabellini Davide | Zendy; Schmidt Uli | Zendy

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A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy‐Affected Skeletal Muscles

Author(s) -

Caron Leslie,

Kher Devaki,

Lee Kian Leong,

McKernan Robert,

Dumevska Biljana,

Hidalgo Alejandro,

Li Jia,

Yang Henry,

Main Heather,

Ferri Giulia,

Petek Lisa M.,

Poellinger Lorenz,

Miller Daniel G.,

Gabellini Davide,

Schmidt Uli

Publication year - 2016

Publication title -

stem cells translational medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.781

H-Index - 71

eISSN - 2157-6580

pISSN - 2157-6564

DOI - 10.5966/sctm.2015-0224

Subject(s) - facioscapulohumeral muscular dystrophy , induced pluripotent stem cell , muscular dystrophy , stem cell , skeletal muscle , biology , medicine , bioinformatics , microbiology and biotechnology , anatomy , genetics , embryonic stem cell , gene

This work describes a monolayer system to differentiate human pluripotent stem cells (hPSCs) into skeletal muscle cells (SkMCs) and demonstrates disease‐specific phenotypes in SkMCs derived from hPSCs affected with facioscapulohumeral muscular dystrophy. This is the first human stem cell‐based cellular model for a muscular dystrophy suitable for high‐throughput screening and drug development.

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