Disease exacerbation in human DMD MYOrganoids enables gene therapy evaluation and unveils persistence of fibrotic activity

Abstract Leading gene therapy approaches for Duchenne muscular dystrophy (DMD) using AAV-mediated delivery of microdystrophin (µDys) have shown partial efficacy in patients, contrasting with the favorable outcomes observed in animal models. The identification of effective therapeutic strategies coul...

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Autori principali:	Laura Palmieri, Giorgia Bimbi, Maxime Ferrand, Matteo Marcello, Louna Pili, Ai Vu Hong, Abbass Jaber, Riyad El-Khoury, Guy Brochier, Anne Bigot, David Israeli, Isabelle Richard, Sonia Albini
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Nature Portfolio 2026-01-01
Serie:	npj Regenerative Medicine
Accesso online:	https://doi.org/10.1038/s41536-025-00445-8
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