Krivit W, Sung J H, Shapiro E G, Lockman L A
Department of Pediatrics, University of Minnesota Medical School, Minneapolis 55455, USA.
Cell Transplant. 1995 Jul-Aug;4(4):385-92. doi: 10.1177/096368979500400409.
Treatment and potential cure of lysosomal and peroxisomal diseases, heretofore considered fatal, has become a reality during the past decade. Bone marrow transplantation, (BMT), has provided a method for replacement of the disease-causing enzyme deficiency. Cells derived from the donor marrow continue to provide enzyme indefinitely. Several scores of patients with diseases as diverse as metachromatic leukodystrophy, adrenoleukodystrophy, globoid cell leukodystrophy, Hurler syndrome (MPS I-H), Maroteaux-Lamy (MPS VI) Gaucher disease, and fucosidosis have been successfully treated following long-term engraftment. Central nervous system (CNS) manifestations are also prevented or ameliorated in animal models of these diseases following engraftment from normal donors. The microglial cell system has been considered to be the most likely vehicle for enzyme activity following bone marrow engraftment. Microglia in the mature animal or human are derived from the newly engrafted bone marrow. Graft-v-host disease activation of the microglia is also of importance. This article will summarize some of the pertinent literature relative to the role of microglia in such transplant processes.
溶酶体和过氧化物酶体疾病的治疗及潜在治愈方法，在过去一直被认为是致命的，但在过去十年中已成为现实。骨髓移植（BMT）提供了一种替代致病酶缺乏的方法。来自供体骨髓的细胞会持续无限期地提供酶。几十名患有多种疾病的患者，如异染性脑白质营养不良、肾上腺脑白质营养不良、球状细胞脑白质营养不良、Hurler综合征（MPS I-H）、Maroteaux-Lamy综合征（MPS VI）、戈谢病和岩藻糖苷贮积症，在长期植入后已成功得到治疗。在这些疾病的动物模型中，从正常供体植入后，中枢神经系统（CNS）表现也得到了预防或改善。小胶质细胞系统被认为是骨髓植入后酶活性最可能的载体。成熟动物或人类中的小胶质细胞源自新植入的骨髓。小胶质细胞的移植物抗宿主病激活也很重要。本文将总结一些与小胶质细胞在这种移植过程中的作用相关的重要文献。
