title: New therapeutic approaches to mendelian disorders.
authors.affiliation: McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, 733 N. Broadway St., Baltimore, MD 21205, USA. hdietz@jhmi.edu
authors.forename: Harry C
authors.initials: HC
authors.lastname: Dietz
mesh_terms: D000818:Animals; D056947:Enzyme Replacement Therapy; D015870:Gene Expression; D030342:Genetic Diseases, Inborn / Q000628:therapy*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008382:Marfan Syndrome / Q000235:genetics / Q000628:therapy; D009154:Mutation; D011371:Progeria / Q000235:genetics / Q000628:therapy; D011506:Proteins / Q000235:genetics; D012322:RNA Precursors / Q000378:metabolism; D012326:RNA Splicing
chemical_list: D011506:Proteins; D012322:RNA Precursors