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骨髓移植是根治白血病、重症再生障碍性贫血及某些遗传性疾病的重要手段。异基因骨髓移植的优点是显而易见的,但它面临移植物排斥,移植物抗宿主病(GVHD)及供者来源等问题。随着分子生物学的发展,基因转移配合自体骨髓移植为遗传性疾病的基因治疗提供了新的可能性。此外耐药基因转移入骨髓造血干细胞,亦将为肿瘤和白血病化疗开辟前景。一、遗传性疾病的分类与骨髓移植选择根据临床表现及异基因骨髓移植的疗效可将遗传性疾病分成3大类。
Bone marrow transplantation is an important means to cure leukemia, severe aplastic anemia and some hereditary diseases. The advantages of allogeneic bone marrow transplantation are obvious, but it faces the problems of graft rejection, graft versus host disease (GVHD) and donor source. With the development of molecular biology, gene transfer and autologous bone marrow transplantation provide a new possibility for gene therapy of hereditary diseases. In addition, resistance gene transfer into bone marrow hematopoietic stem cells, will also open up prospects for the treatment of cancer and leukemia. First, the classification of genetic diseases and bone marrow transplantation options According to the clinical manifestations and allogeneic bone marrow transplantation can be divided into three major categories of genetic diseases.