Beutler E
Department of Molecular and Experimental Medicine, Scripps Research Institute, La Jolla, California.
West J Med. 1994 Feb;160(2):129-32.
Marrow transplantation has proved to be an important modality in the treatment of aplastic anemia, acute leukemia, and some other malignant diseases of hemopoietic cells. Much less attention has been paid to the role of marrow transplantation in the treatment of stem cell disorders such as sickle cell disease, chronic granulomatous disease, or Gaucher's disease. Allogeneic transplantation is successful in these disorders, but carries a considerable risk to the recipient. Autologous transplantation becomes a reality when efficient gene transfer with sustained expression is developed. As methods are developed to harvest hemopoietic stem cells from the peripheral blood and to amplify these cells without causing them to differentiate, transplantation will become increasingly valuable in the treatment of stem cell disorders.
骨髓移植已被证明是治疗再生障碍性贫血、急性白血病及其他一些造血细胞恶性疾病的重要手段。然而，骨髓移植在治疗诸如镰状细胞病、慢性肉芽肿病或戈谢病等干细胞疾病中的作用却很少受到关注。同种异体移植在这些疾病中是成功的，但对接受者有相当大的风险。当高效基因转移并能持续表达得以实现时，自体移植就成为了现实。随着从外周血中采集造血干细胞并在不使其分化的情况下扩增这些细胞的方法不断发展，移植在干细胞疾病治疗中将变得越来越有价值。
