[Gene therapy for genetic disorders of purine metabolism].
Anderson proposed three genetic diseases deficient in enzymes involved in purine metabolism as candidate for human gene therapy in 1984. Over the past ten years, marked advances in molecular genetics have brought gene therapy to reality. Gene therapy for ADA deficiency was performedin the United States in 1990 and in Japan in 1995 as the first trial. Basic study for HPRT gene transfer has been proceeding toward gene therapy for HPRT deficiency despite the difficulties in gene transfer into the nervous systems.
Hidaka
Y
Y
Teikyo University School of Medicine, Third Department of Internal Medicine.
D000243:Adenosine Deaminase / Q000172:deficiency / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D006801:Humans; D007041:Hypoxanthine Phosphoribosyltransferase / Q000172:deficiency / Q000235:genetics; D011686:Purine-Pyrimidine Metabolism, Inborn Errors / Q000235:genetics / Q000628:therapy*
D007041:Hypoxanthine Phosphoribosyltransferase; D000243:Adenosine Deaminase