title: Gene therapy of metabolic diseases.
abstract: This review surveys the potential application of gene therapy to metabolic diseases. Proof of principle has been achieved in the treatment of two inherited immunodeficiency conditions. A significant safety issue has also been observed. Several strategies are being experimentally tested for a number of metabolic diseases (more than 20) and may provide a rationale for the future development of clinical trials.
authors.affiliation: Inserm, U768, Paris, France. fischer@necker.fr
authors.forename: Alain
authors.initials: A
authors.lastname: Fischer
authors.forename: Salima
authors.initials: S
authors.lastname: Hacein-Bey-Abina
authors.forename: Marina
authors.initials: M
authors.lastname: Cavazzana-Calvo
mesh_terms: D018390:Gene Targeting; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000009:adverse effects; D006801:Humans; D008661:Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy*; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D016896:Treatment Outcome