title: Adrenoleukodystrophy patient perspective: turning despair into a gene therapy breakthrough.
authors.forename: Amber
authors.initials: A
authors.lastname: Salzman
mesh_terms: D000074063:ATP Binding Cassette Transporter, Subfamily D, Member 1; D018528:ATP-Binding Cassette Transporters / Q000235:genetics; D000326:Adrenoleukodystrophy / Q000235:genetics* / Q000628:therapy*; D035843:Biomedical Research; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends*; D006801:Humans; D033581:Stem Cell Transplantation
chemical_list: C115572:ABCD1 protein, human; D000074063:ATP Binding Cassette Transporter, Subfamily D, Member 1; D018528:ATP-Binding Cassette Transporters