Open access
Aug 2026
Advanced iPSC-based modelling of LMNA-related congenital muscular dystrophy enables development of genetic therapies for muscle laminopathies.
The selection of L-CMD iPSCs is expanded, disease-associated readouts are validated using a transgene-free differentiation protocol and gene editing strategies are assessed using 2D and 3D cultures, providing an advanced, humanised platform for translational research and precision medicine in laminopathies.
D. Moore, H. Steele-Stallard, L. Pinton et al.
· Molecular Therapy · 0 citations