Muscle-specific gene editing improves molecular and phenotypic defects in a mouse model of myotonic dystrophy type 1

Izzo M, Battistini J, Golini E, Voellenkle C, Provenzano C, Orsini T, Strimpakos G, Scavizzi F, Raspa M, Baci D, Frolova S, Tastsoglou S, Zaccagnini G, Garcia-Manteiga JM, Gourdon G, Mandillo S, Cardinali B, Martelli F, Falcone G

Clinical and translational medicine