[The gene therapy for a patient with ADA deficiency; report of the first gene therapy trial in Japan].
Since the first gene therapy clinical trial for an ADA deficient patient was performed in September 1990, 10 ADA deficient patients have been enrolled in gene therapy clinical trial. We have been performing the first gene therapy trial in Japan for a 5 year boy with ADA deficiency since August 1995. Activated T cells from the patient's peripheral mononuclear cells were transduced by a retrovirus vector, LASN, which contained cDNA of human ADA gene, and re-infused to him intravenously after 7-11 days. We have already performed 10 cycles of the therapy for the patient. Here, we report the successful results of the gene therapy with laboratory and clinical evaluation. Furthermore, we overview the results of gene therapy for ADA deficient patients which were recently reported from 4 other groups.
Ariga
T
T
Department of Pediatrics, Hokkaido University School of Medicine.
Kawamura
N
N
Sakiyama
Y
Y
D000243:Adenosine Deaminase / Q000172:deficiency* / Q000235:genetics; D002675:Child, Preschool; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008297:Male; D012190:Retroviridae; D016511:Severe Combined Immunodeficiency / Q000628:therapy*
D000243:Adenosine Deaminase