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GENE THERAPY FOR X-LINKED SEVERE COMBINED IMMUNODEFICIENCY DISEASE

X连锁严重联合免疫缺陷病的基因治疗

基本信息

批准号：
14370240
负责人：
TSUCHIYA Shigeru
金额：
$ 9.02万
依托单位：
TOHOKU UNIVERSITY
依托单位国家：
日本
项目类别：
Grant-in-Aid for Scientific Research (B)
财政年份：
2002
资助国家：
日本
起止时间：
2002 至 2003
项目状态：
已结题

项目摘要

(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.

(1)X-连锁重症联合免疫缺陷（X-SCID）的基因治疗由于111例接受X-SCID基因治疗的患者中有2例发生白血病样疾病，该计划一直悬而未决。我们将重新考虑X-SCID基因治疗的新适应症。(2)对1例X-SCID患者进行了HLA单倍型相合的外周血干细胞移植，并对患者进行了免疫功能的随访研究。他的免疫功能很快恢复，没有任何移植物抗宿主病的证据。(3)非清髓性骨髓移植（NST）治疗慢性肉芽肿性疾病（CGD）1例18岁的CGD患者。（4）IL-7受体α链缺陷病我们在亚洲首例有B淋巴细胞的SCID患者中发现了IL-7 R α缺陷病。

项目成果

期刊论文数量（46）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

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)

Kudo Y 等人：“外周血 0034 细胞的绝对数量可预测血液恶性肿瘤和实体瘤患者的单采血液成分和祖细胞产量的时机”Tohoku J Exp Med。

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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)

Shigeru Tsuchiya：“使用逆转录病毒载体进行基因治疗的白血病”BIO Clinica 18. 80-85 (2003)。

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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)

Itoh T 等人：“一种无需速率控制程序冷冻机的简单的速率控制冷冻方法，为脐带血细胞的大规模和小规模冷冻保存提供了最佳条件”。

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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. 348-3

Kobayashi N、Agematsu K、Sugita K、Sako M、Nonoyama S、Yachie A、Kumaki S、Tsuchiya S、Ochs HD、Sugita K、Fukushima Y、Komiyama A：“日本家庭中放射敏感性严重联合免疫缺陷的新型 Artemis 基因突变”