title: Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders.
abstract: Many metabolic diseases are compelling candidates for gene therapy, and are the subject of vigorous pre-clinical research. Successful phenotype correction in mouse models is now commonplace and research effort is increasingly being directed towards addressing the translational challenges inherent in human clinical trials. This paper places current efforts to develop gene therapy approaches to metabolic disease in historical context and describes contemporary research in the authors' laboratory on urea cycle defects, particularly ornithine transcarbamylase deficiency, in a manner that is illustrative of the general state of the field.
authors.affiliation: Gene Therapy Research Unit, Children's Medical Research Institute and The Children's Hospital at Westmead, Locked Bag 4001, Westmead, NSW, 2145, Australia. ian.alexander@health.nsw.gov.au
authors.forename: Ian E
authors.initials: IE
authors.lastname: Alexander
authors.forename: Cindy
authors.initials: C
authors.lastname: Kok
authors.forename: Allison P
authors.initials: AP
authors.lastname: Dane
authors.forename: Sharon C
authors.initials: SC
authors.lastname: Cunningham
mesh_terms: D000818:Animals; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008659:Metabolic Diseases / Q000235:genetics* / Q000628:therapy*; D051379:Mice; D056806:Urea Cycle Disorders, Inborn / Q000235:genetics* / Q000628:therapy*