MICROGLIA - THE EFFECTOR CELL FOR RECONSTITUTION OF THE CENTRAL-NERVOUS-SYSTEM FOLLOWING BONE-MARROW TRANSPLANTATION FOR LYSOSOMAL AND PEROXISOMAL STORAGE DISEASES

Treatment and potential cure of lysosomal and peroxisomal diseases, he retofore considered fatal, has become a reality during the past decade . Bone marrow transplantation, (BMT), has provided a method for replac ement 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 (MP S I-H), Maroteaux-Lamy (MPS VI) Gaucher disease, and fucosidosis have been successfully treated following long-term engraftment. Central ner vous system (CNS) manifestations are also prevented or ameliorated in animal models of these diseases following engraftment from normal dono rs. The microglial cell system has been considered to be the most like ly vehicle for enzyme activity following bone marrow engraftment. Micr oglia in the mature animal or human are derived from the newly engraft ed bone marrow. Graft-v-host disease activation of the microglia is al so of importance. This article will summarize some of the pertinent li terature relative to the role of microglia in such transplant processe s.