Prednisolone rescues Duchenne muscular dystrophy phenotypes in human pluripotent stem cell-derived skeletal muscle in vitro.

doi:10.1073/pnas.2022960118

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Article

Prednisolone rescues Duchenne muscular dystrophy phenotypes in human pluripotent stem cell-derived skeletal muscle in vitro.

Proceedings of the National Academy of Sciences of the United States of America - 13 Jul 2021

Al Tanoury Ziad, Zimmerman John F, Rao Jyoti, Sieiro Daniel, McNamara Harold M, Cherrier Thomas, Rodríguez-delaRosa Alejandra, Hick-Colin Aurore, Bousson Fanny, Fugier-Schmucker Charlotte, Marchiano Fabio, Habermann Bianca, Chal Jérome, Nesmith Alexander P, Gapon Svetlana, Wagner Erica, Gupta Vandana A, Bassel-Duby Rhonda, Olson Eric N, Cohen Adam E, Parker Kevin Kit, Pourquié Olivier

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 us to recapitulate classical DMD phenotypes (mislocalization of proteins of the...

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Identifiers

PMID: 34260377
DOI: 10.1073/pnas.2022960118
PMCID: PMC8285911

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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