title: Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy.
abstract: Transgene SA is developing Myodys, a non-viral plasmid gene therapy for the potential treatment of Duchenne muscular dystrophy and Becker muscular dystrophy. Phase I clinical trials have been completed, and a phase II clinical trial is planned.
authors.affiliation: University of Missouri, Department of Molecular Microbiology and Immunology, M610 Medical Sciences Building, One Hospital Drive, Columbia, MO 65212, USA. duand@missouri.edu
authors.forename: Dongsheng
authors.initials: D
authors.lastname: Duan
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D000075202:Contraindications; D004353:Drug Evaluation, Preclinical; D016189:Dystrophin / Q000235:genetics* / Q000627:therapeutic use*; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006801:Humans; D051379:Mice; D020388:Muscular Dystrophy, Duchenne / Q000235:genetics* / Q000628:therapy*; D010330:Patents as Topic; D010957:Plasmids / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D013329:Structure-Activity Relationship
chemical_list: D016189:Dystrophin