Cécile Peccate

This study shows that axonopathy in DMD limits microdystrophin gene therapy efficacy by demonstrating that dystrophin is expressed in peripheral nerves and its absence causes nerve structural collapse and impaired nerve-muscle connectivity.

This study identifies NOTCH3 and GLIS3 as contributors to regeneration impairment in Duchenne Muscular Dystrophy using spatial transcriptomics and multiomic profiling.

This study identifies Loxl2 as a therapeutic target for LMNA cardiomyopathy by showing its role in ECM remodeling in patient-derived cardiomyocytes and mouse models.