Zintl F, Hermann J, Fuchs D, Prager J, Müller A, Reiners B, Füller J
Abteilung für Hämatologie, Onkologie und Immunologie, Universitäts-Kinderklinik Jussuf Ibrahim.
Kinderarztl Prax. 1991 Jan-Feb;59(1-2):6-9.
Bone marrow transplantation (BMT) was mainly used for the treatment of immunological, hematological and oncological diseases. Genetic diseases can also be treated by transplantation of normal histocompatible allogeneic bone marrow. This type of marrow transplantation is used for the treatment of inborn errors whose clinical expression is restricted to lymphoid or hematopoietic cells. The therapeutic role of bone marrow transplantation in the treatment of generalized genetic diseases is under investigation. Genetic disorders that can be cured include congenital immune deficiencies, infantile malignant osteopetrosis, thalassemia, infantile agranulocytosis, chronic granulomatous disease, lysosomal storage diseases and others. The use of autologous bone marrow after the insertion of a normal gene (gene therapy) in vitro circumvents the need for a histocompatible donor.
骨髓移植（BMT）主要用于治疗免疫、血液和肿瘤疾病。遗传性疾病也可通过移植正常组织相容性同种异体骨髓进行治疗。这种类型的骨髓移植用于治疗临床表现局限于淋巴细胞或造血细胞的先天性代谢缺陷病。骨髓移植在治疗全身性遗传性疾病中的治疗作用正在研究中。可以治愈的遗传性疾病包括先天性免疫缺陷、婴儿恶性骨硬化症、地中海贫血、婴儿粒细胞缺乏症、慢性肉芽肿病、溶酶体贮积病等。在体外插入正常基因（基因治疗）后使用自体骨髓，避免了对组织相容性供体的需求。
