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GENE THERAPY FOR X-LINKED SEVERE COMBINED IMMUNODEFICIENCY DISEASE

Research Project

Project/Area Number 14370240
Research Category

Grant-in-Aid for Scientific Research (B)

Allocation TypeSingle-year Grants
Section一般
Research Field Pediatrics
Research InstitutionTOHOKU UNIVERSITY

Principal Investigator

TSUCHIYA Shigeru  TOHOKU UNIVERSITY, INSUTITUTE OF AGING, DEVELOPMENT AND CANCR, PROFESSOR, 加齢医学研究所, 教授 (30124605)

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)
Project Period (FY) 2002 – 2003
Project Status Completed (Fiscal Year 2003)
Budget Amount *help
¥14,100,000 (Direct Cost: ¥14,100,000)
Fiscal Year 2003: ¥4,000,000 (Direct Cost: ¥4,000,000)
Fiscal Year 2002: ¥10,100,000 (Direct Cost: ¥10,100,000)
KeywordsX-linked Severe combined immunodeficiency / gene therapy / hematopoietic stem cell transplantation / non-myeloablative / haplotype identical / IL-'7receptor deficiency disease / X連鎖 / 重症複合免疫症 / HLAハプロタイプ一致 / 純化CD34陽性細胞
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.

Report

(3 results)
  • 2003 Annual Research Report   Final Research Report Summary
  • 2002 Annual Research Report
  • Research Products

    (23 results)

All Other

All Publications (23 results)

  • [Publications] Itoh T et al.: "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)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2003 Final Research Report Summary
  • [Publications] Kobayashi N et al.: "Novel Artemis gene mutations of radiosensitive severe combined immunodeficiency in Japanese families."Hum Genet.. 112. 348-352 (2003)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2003 Final Research Report Summary
  • [Publications] Imashuku S et al.: "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)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2003 Final Research Report Summary
  • [Publications] Kimura H et al.: "Prognostic factors for chronic active Epstein-Barr virus infection."J Infect Dis. 187. 527-533 (2003)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2003 Final Research Report Summary
  • [Publications] Kudo Y et al.: "The absolute number of peripheral blood 0034+ 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)

    • Description
      「研究成果報告書概要(和文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [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)

    • Description
      「研究成果報告書概要(欧文)」より
    • Related Report
      2003 Final Research Report Summary
  • [Publications] Itoh T et al.: "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)

    • Related Report
      2003 Annual Research Report
  • [Publications] Kobayashi N et al.: "Novel Artemis gene mutations of radiosensitive severe combined immunodeficiency in Japanese families"Hum Genet.. 112. 348-352 (2003)

    • Related Report
      2003 Annual Research Report
  • [Publications] Imashuku S et al.: "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)

    • Related Report
      2003 Annual Research Report
  • [Publications] Kimura H et al.: "Prognostic factors for chronic active Epstein-Barr virus infection"J Infect Dis. 187. 527-533 (2003)

    • Related Report
      2003 Annual Research Report
  • [Publications] Kudo Y et al.: "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)

    • Related Report
      2003 Annual Research Report
  • [Publications] 土屋 滋: "レトロウイルスベクターを用いた遺伝子治療による白血病"BIO Clinica. 18. 80-85 (2003)

    • Related Report
      2003 Annual Research Report
  • [Publications] Kawai et al.: "Flow cytometric determination of intracytoplasmic Wiskott-Aldrich syndromeprotein in peripheral blood lymphocyte subpopulations"J. Immunol Methods. 260. 195-205 (2002)

    • Related Report
      2002 Annual Research Report
  • [Publications] Tsuchiya S.: "Diagnosis of Epstein-Barr virus-associated diseases"Crit Rev. Oncol Hematol.. 44. 227-238 (2002)

    • Related Report
      2002 Annual Research Report
  • [Publications] Ohga et al.: "Treatment responses of childhood aplastic anaemia with chromosomal aberrations at diagnosis"British J. Haematology. 118. 313-319 (2002)

    • Related Report
      2002 Annual Research Report
  • [Publications] 土屋 滋: "X連鎖重症複合免疫不全症の遺伝子治療"日本小児血液学会雑誌. 16. 2-10 (2002)

    • Related Report
      2002 Annual Research Report
  • [Publications] 土屋 滋: "小児の遺伝子治療"小児科. 43. 1872-1879 (2002)

    • Related Report
      2002 Annual Research Report
  • [Publications] 土屋 滋: "CD45の異常と重症複合免疫不全症"臨床免疫. 38. 653-658 (2002)

    • Related Report
      2002 Annual Research Report

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Published: 2002-04-01   Modified: 2016-04-21  

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