Advanced human iPSC-based modelling of LMNA-related congenital muscular dystrophy enables development of targeted genetic therapies for muscle laminopathies
Daniel Moore, Heather B. Steele-Stallard, Luca Pinton, Valentina Maria Lionello, Lucia Rossi, Artadokht Aghaeipour, Salma Jalal, Cherry Tsz Yan Wong, Angela Clara-Hwang, Gisèle Bonne, Peter S. Zammit, Francesco Saverio Tedesco
This paper develops a human iPSC-based model of L-CMD to test gene editing strategies for muscle laminopathies, showing partial reversal of disease phenotypes with precise mutation correction.
bioRxiv · Jun 22, 2025 · PreprintCell Biology