Article
Prednisolone rescues Duchenne Muscular Dystrophy phenotypes in human pluripotent stem cells-derived skeletal muscle <i>in vitro</i>
2020-10-29
Abstract excerpt
Duchenne Muscular Dystrophy (DMD) is a devastating genetic disease leading to degeneration of skeletal muscles and premature death. How dystrophin absence leads to muscle wasting remains unclear. Here, we describe an optimized protocol to differentiate human induced Pluripotent Stem Cells (iPSC) to a late myogenic stage. This allows to recapitulate classical DMD phenotypes (mislocalization of proteins of the Dystr...
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Identifiers and source
- Literature Corpus work
- e44ec154-179b-583a-87db-ec576a66b8b9
- DOI
- 10.1101/2020.10.29.360826
