A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy-Affected Skeletal Muscles
Summary
This work describes an efficient and highly scalable monolayer system to differentiate human pluripotent stem cells (hPSCs) into skeletal muscle cells (SkMCs) and demonstrates disease-specific phenotypes in SkMCs derived from both embryonic and induced hPSCs affected with facioscapulohumeral muscular dystrophy. This study represents the first human stem cell-based cellular model for a muscular dystrophy that is suitable for high-throughput screening and drug development. ©AlphaMed Press.
| Authors | Caron L, Kher D, Lee KL, McKernan R, Dumevska B, Hidalgo A, Li J, Yang H, Main H, Ferri G, Petek LM, Poellinger L, Miller DG, Gabellini D, Schmidt U |
|---|---|
| Journal | Stem cells translational medicine |
| Publication Date | 2016 Sep;5(9):1145-61 |
| PubMed | 27217344 |
| PubMed Central | PMC4996435 |
| DOI | 10.5966/sctm.2015-0224 |