Annemieke Aartsma‐Rus

Four new Duchenne muscular dystrophy mouse models with clinically relevant exon deletions in the human DMD gene

Abstract Mutation specific therapeutic approaches, like exon skipping or gene-editing, hold promise for the treatment of Duchenne muscular dystrophy (DMD). Translatability of preclinical studies investigating these approaches could greatly be improved through the use of humanized...