2003 Fiscal Year Final Research Report Summary
GENE THERAPY FOR X-LINKED SEVERE COMBINED IMMUNODEFICIENCY DISEASE
| Project/Area Number |
14370240
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | TOHOKU UNIVERSITY |
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Principal Investigator |
TSUCHIYA Shigeru TOHOKU UNIVERSITY, INSUTITUTE OF AGING, DEVELOPMENT AND CANCR, PROFESSOR, 加齢医学研究所, 教授 (30124605)
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| Co-Investigator(Kenkyū-buntansha) |
ANAN Kazuaki TOHOKU UNIVERSITY, INSTITUTE OF AGING, DEVELPMENT AND CANCERAND CANCER, ASSISTANT, 加齢医学研究所, 助手 (80343036)
MINEGISHI Masayoshi TOHOKU UNIVERSITY HOSPITAL, ASSOCIATE PROFESSOR, 医学部附属病院, 助教授 (20211592)
KUMAKI Satoru MTOHOKU UNIVERSITY, INSTITUTE OF AGING, DEVELOPMENT AND CANCER, ASSOCIATE PROFESSOR, 加齢医学研究所, 助教授 (20311566)
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| Project Period (FY) |
2002 – 2003
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| Keywords | X-linked Severe combined immunodeficiency / gene therapy / hematopoietic stem cell transplantation / non-myeloablative / haplotype identical / IL-'7receptor deficiency disease |
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| Research Abstract |
(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-SCID
CD34 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 disease
We found IL-7R α deficiency disease among patients with SCID with B lymphocytes, the first case in asia.
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Research Products
(11 results)
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[Publications] Itoh T, Minegishi M, Fushimi J, Takahashi H, Kudo Y, Suzuki A, Narita A, Sato Y, Akagi K, Wada Y, Saito A, Kikuchi M, Okamura K, Kaku M, Tsuchiya S.: "A simple controlled-rate freezing method without a rate-controlled programmed freezer provides optimal conditions for both large-scale and small-scale cryopreservation of umbilical cord blood cells"Transfusion. 43. 1303-1308 (2003)
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[Publications] Kobayashi N, Agematsu K, Sugita K, Sako M, Nonoyama S, Yachie A, Kumaki S, Tsuchiya S, Ochs HD, Sugita K, Fukushima Y, Komiyama A: "Novel Artemis gene mutations of radiosensitive severe combined immunodeficiency in Japanese families"Hum Genet.. 112. 348-352 (2003)
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[Publications] Imashuku S, Terui K, Matsuyama T, Asami K, Tsuchiya S, Ishii E, Kawa K, Kosaka Y, Eguchi H, Tsuchida M, Ikuta K, Kato S, Koizumi S, Okamura J, Morimoto A, Hibi S, Hamaoka K: "Multi-institutional collaborative study in Japan. Lack of clinical utility of minimal residual disease detection in allogeneic stem cell recipients with childhood acute lymphoblastic leukemia : multi-institutional collaborative study in Japan"Bone Marrow Transplant. 31. 1127-1135 (2003)
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「研究成果報告書概要(欧文)」より
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[Publications] Kimura H, Morishima T, Kanegane H, Ohga S, Hoshino Y, Maeda A, Imai S, Okano M, Morio T, Yokota S, Tsuchiya S, Yachie A, Imashuku S, Kawa K, Wakiguchi H: "Japanese Association for Research on Epstein-Barr Virus and Related Diseases : Prognostic factors for chronic active Epstein-Barr virus infection"J Infect Dis. 187. 527-533 (2003)
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「研究成果報告書概要(欧文)」より
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[Publications] Kimura H, Morishima T, Kanegane H, Ohga S, Hoshino Y, Maeda A, Imai S, Okano M, Morio T, Yokota S, Tsuchiya S, Yachie A, Imashuku S, Kawa K, Wakiguchi H: "Japanese Association for Research on Epstein-Barr Virus and Related Diseases : Prognostic factors for chronic active Epstein-Barr virus infection"J Infect Dis. 118. 313-319 (2002)
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[Publications] Kudo Y, Minegishi M, Saito N, Itoh T, Fushimi J, Takahashi H, Suzuki A, Narita A, Sato Y, Miura J, Yoshihara K, Meguro K, Imaizumi M, Sato M, Murakawa Y, Tsuchiya S.: "The Absolute Number of Peripheral Blood CD34+ Cells Predicts a Timing for Apheresis and Progenitor Cell Yield in Patients with Hematologic Malignancies and Solid Tumors"Tohoku J Exp Med.. 199. 111-118 (2003)
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