title: Leukodystrophy and gene therapy with a dimmer switch.
authors.affiliation: Department of Pediatrics and the Bone and Marrow Transplant Program, University of Minnesota Medical School, Minneapolis, USA.
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Orchard
authors.forename: John E
authors.initials: JE
authors.lastname: Wagner
mesh_terms: D005698:Galactosylceramidase / Q000235:genetics* / Q000378:metabolism; D005786:Gene Expression Regulation; D015316:Genetic Therapy; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D006801:Humans; D007965:Leukodystrophy, Globoid Cell / Q000235:genetics / Q000628:therapy*; D035683:MicroRNAs
chemical_list: D035683:MicroRNAs; D005698:Galactosylceramidase