title: Gene therapy in man and mice: adenosine deaminase deficiency, ornithine transcarbamylase deficiency, and Duchenne muscular dystrophy.
authors.affiliation: Institute for Molecular Genetics, Baylor College of Medicine, Houston, TX.
authors.forename: M
authors.initials: M
authors.lastname: Grompe
authors.forename: K
authors.initials: K
authors.lastname: Mitani
authors.forename: C C
authors.initials: CC
authors.lastname: Lee
authors.forename: S N
authors.initials: SN
authors.lastname: Jones
authors.forename: C T
authors.initials: CT
authors.lastname: Caskey
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency* / Q000235:genetics; D000592:Amino Acid Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy; D000818:Animals; D016189:Dystrophin / Q000235:genetics; D015316:Genetic Therapy; D006801:Humans; D008297:Male; D051379:Mice; D009137:Muscular Dystrophy, Animal / Q000235:genetics / Q000628:therapy*; D009954:Ornithine Carbamoyltransferase / Q000235:genetics; D020163:Ornithine Carbamoyltransferase Deficiency Disease; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy
chemical_list: D016189:Dystrophin; D009954:Ornithine Carbamoyltransferase; D000243:Adenosine Deaminase