title: Gene Therapy for Metabolic Diseases.
abstract: Gene therapy has recently shown great promise as an effective treatment for a number of metabolic diseases caused by genetic defects in both animal models and human clinical trials. Most of the current success has been achieved using a viral mediated gene addition approach, but gene-editing technology has progressed rapidly and gene modification is being actively pursued in clinical trials. This review focuses on viral mediated gene addition approaches, because most of the current clinical trials utilize this approach to treat metabolic diseases.
authors.affiliation: Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
authors.forename: Randy J
authors.initials: RJ
authors.lastname: Chandler
authors.affiliation: Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
authors.forename: Charles P
authors.initials: CP
authors.lastname: Venditti
keywords: Gene Delivery; Gene Therapy; Genomic Medicine; Inborn Errors of Metabolism; Metabolic Disorders; Therapy for Rare Disease