Researchers at Queen Mary University of London and University College London have shown that using stem cells derived from patients could help find new treatments for a group of congenital muscular dystrophies.
The proof of concept study generated the first patient-derived induced pluripotent stem cell (iPSC) model for studying dystroglycanopathy – a group of congenital muscular dystrophies often associated with brain malformations.
“Our ability to test potential drugs on people with one of these conditions has been hampered by the lack of appropriate human cell models. We are confident that this study overcomes that challenge.”
