[Study of T cell-directed gene transfer for a patient with ADA deficiency].
We have been undertaking the gene therapy for a 6 year-old boy with ADA deficiency and performed 11 cycles of the infusion of the peripheral T cells transduced with retroviral vector LASN since August 8th 1995. The percentage of the peripheral blood lymphocytes carrying the transduced ADA gene has remained stable at 10% to 20% since the 4th infusion. ADA enzyme activity in his circulating T cells increased to levels comparable to 1/3 of a heterozygous carrier individual and was associated with increased T lymphocytes counts and improvement in both humoral and cellular immune function. The results obtained in this clinical study support the usefulness of T lymphocyte-directed gene transfer in the treatment of ADA deficiency.
Sakiyama
Y
Y
Department of Pediatrics, Hokkaido University School of Medicine.
Ariga
T
T
Kawamura
N
N
D000243:Adenosine Deaminase / Q000172:deficiency* / Q000235:genetics; D002648:Child; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D008297:Male; D013601:T-Lymphocytes
D000243:Adenosine Deaminase