BioengineeringbioRxivPreprint
Patient-derived 3D engineered human muscle model recapitulates CLCN1 mis-splicing and myotonia in myotonic dystrophy type 1
Xiomara Fernández‐Garibay, María Sabater-Arcis, Ainoa Tejedera‐Villafranca…·Juan M. Fernández‐Costa
This paper develops a 3D human muscle model from DM1 patient myoblasts that recapitulates CLCN1 mis-splicing and myotonia, and tests therapeutic responses.
DOI 10.64898/2026.02.03.703003