Human iPSC-based model for Duchenne muscular dystrophy

ABSTRACT: We developed a novel somite-based step-wise strategy for the efficient derivation of functional human myocytes, suggesting that past failures were due to incomplete specification. Treatment with two small molecules inhibiting glycogen synthase kinase 3β (GSK-3β) and the Notch signaling pathway in undifferentiated hPSCs results in the formation of somite-like cells by Day 4 and, subsequently contractile myotubes in vitro around Day 25 with the ability to engraft and actively participate in muscle repair in vivo. Antibody-based purification can enrich homogenous myocyte populations exhibiting genuine myogenic molecular and cellular characteristics, including extraocular muscle-like features. Furthermore, hPSCs derived from patients with multiple neuromuscular diseases successfully give ris

ORGANISM(S): Homo sapiens

SUBMITTER: Gabsang Lee 

PROVIDER: E-GEOD-70955 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress