GENE THERAPY FOR X-LINKED SEVERE COMBINED IMMUNODEFICIENCY DISEASE

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(1) Gene therapy for X-linked severe combined immunodeficiency (X-SCID)Since two cases of leukemia like disease were reported from A Fischer's group among 11patients who received X-SCID gene therapy, the plan was now kept in suspense. We are going to reconsider the X-SCID gene therapy with new indications of the therapy.(2) HLA haplotype identical peripheral stem cell transplantation to a patient with X-SCIDCD34 purified peripheral stem cell transplantation from the HLA haplotype identical mother were successfully carried out to the patient with XSCID and put on follow up study of his immune function. His immune function recovered soon without any evidence of graft versus host disease.(3) Non-myeloablative bone marrow transplantation (NST) to a patient with chronic granulomatous diesesase(CGD)NST was successfully performed to an 18-year-old patient with CGD. Chimerism of lymphocytes and granylocytes were achieved soon with recovery of superoxide production in granulocytes.Combination of these two transplantation methods will enable us to establish more safe and efficient tranplantation method for X-SCID,(4) IL-7receptor(R) α chain deficiency diseaseWe found IL-7R α deficiency disease among patients with SCID with B lymphocytes, the first case in asia.