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Study of bone marrow transplantation for the patient with mucopolysaccharidosis.

粘多糖贮积症患者骨髓移植的研究。

基本信息

批准号：
59440047
负责人：
ORII Tadao
金额：
$ 7.04万
依托单位：
Gifu University
依托单位国家：
日本
项目类别：
Grant-in-Aid for General Scientific Research (A)
财政年份：
1984
资助国家：
日本
起止时间：
1984 至 1985
项目状态：
已结题

项目摘要

1.Studies of bone marrow transplantation (BMT)Two basic studies were carried out in vitro for establishment of BMT.(1) Immunological properties of bone marrow cells treated with soybeen agglutinin and sheep red blood cell rosette methods. Bone marrow cells were bractionated by this method described by Reisner et al. for prevention of GVH, and then immunological properties of this fraction were investigated. In results, the bone marrow fraction obtained by this method had very little mature T cells and had low alloreactivity. These results suggest that the bone marrow fraction obtained by this method is able to be used for BMT.(2) Effects of cyclosporin A (CyA) and methotoxesate (MTX) for mixed lymphocyte reaction (MLR). CyA or MTX was usual used for prevention of BVH in vivo. MLR was one of the parameters for GVH. In this study, suppressive effects of CyA or MTX for MLR were investigated. In results, CyA or MTX greatly suppressed MLR. These results suggested that CyA or MTX is able to be use for prevention of GVH in BMT.2.Clinical trial of fibroblast transplantationOne patients with Mucopolysaccharidosis IH/S and one patient with Gaucher disease were treated by fibroblast transplantation. After therapy, it was recognized that both patients' liver size reduced transiently, but there was no effect on the other clinical picture. We conclude that the fibroblast transplantation was not therapeutically useful in these patients.

1.骨髓移植的研究为建立骨髓移植模型，在体外进行了两项基础研究。(1)大豆凝集素和绵羊红细胞花环法处理骨髓细胞的免疫学特性。通过Reisner等描述的用于预防GVH的该方法将骨髓细胞进行支架化，然后研究该级分的免疫学性质。结果，通过该方法获得的骨髓部分具有非常少的成熟T细胞并且具有低同种异体反应性。这些结果表明，通过这种方法获得的骨髓组分能够用于BMT。(2)环孢菌素A（CyA）和甲氨蝶呤（MTX）对混合淋巴细胞反应（MLR）的影响CyA或MTX通常用于体内预防BVH。MLR是GVH的指标之一。在这项研究中，CyA或MTX对MLR的抑制作用进行了研究。结果表明，CyA或MTX显著抑制MLR。结果提示，CyA或MTX可用于预防骨髓移植中GVH的发生。2.成纤维细胞移植的临床试验采用成纤维细胞移植治疗1例粘多糖沉积症IH/S患者和1例戈谢病患者。治疗后，认识到两名患者的肝脏大小短暂减小，但对其他临床表现无影响。我们的结论是，成纤维细胞移植是没有治疗这些患者的有用。