Giorgio A. Tasca

Sep 6, 2023 | nature.com | Daniel Natera |José Verdú-Díaz |Giorgio A. Tasca |Volker Straub |Elisa Villalobos

AbstractDuchenne muscular dystrophy is a genetic disease produced by mutations in the dystrophin gene characterized by early onset muscle weakness leading to severe and irreversible disability. The cellular and molecular consequences of the lack of dystrophin in humans are only partially known, which is crucial for the development of new therapies aiming to slow or stop the progression of the disease.