MBNL-dependent impaired development within the neuromuscular system in myotonic dystrophy type 1.

Julie Tahraoui-BoriesAntoine MérienAnchel González-BarrigaJeanne LainéCéline LeteurHélène PolvècheAlexandre CarteronJuliette Duchesne De LamotteCamille NicoleauJérome PolentesMargot JarrigeMário Gomes-PereiraErwann VentrePauline PoydenotDenis FurlingLaurent SchaefferClaire LegayCécile Martinat

Published in: Neuropathology and applied neurobiology (2023)

Beyond the application to DM1, our approach to generating a robust and reliable human neuromuscular system should facilitate disease modelling studies and drug screening assays.

Keyphrases

endothelial cells
early onset
induced pluripotent stem cells
high throughput
pluripotent stem cells
emergency department
muscular dystrophy
metabolic syndrome