Gene-corrected human iPSC-derived cardiomyocytes and skeletal muscles reveal partial dystrophin Dp427 preservation and cardiac Dp116 expression in Duchenne muscular dystrophy
Marta Białobrzeska, Marta Przymuszała, Anna Potulska-Chromik, Anna Kostera‐Pruszczyk, Jacek Stępniewski, Urszula Florczyk, Józef Dulak
The paper uses hiPSCs to study DMD, finding preserved cardiac dystrophin but reduced skeletal muscle dystrophin and cardiac Dp116 expression.
medRxiv · Jul 11, 2025 · PreprintGenetic & Genomic Medicine