Gene therapy of metabolic diseases.
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.
Fischer
Alain
A
Inserm, U768, Paris, France. fischer@necker.fr
Hacein-Bey-Abina
Salima
S
Cavazzana-Calvo
Marina
M
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