title: Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases.
abstract: 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.
authors.affiliation: Department of Pediatrics, University of Minnesota Medical School, Minneapolis 55455, USA.
authors.forename: W
authors.initials: W
authors.lastname: Krivit
authors.forename: J H
authors.initials: JH
authors.lastname: Sung
authors.forename: E G
authors.initials: EG
authors.lastname: Shapiro
authors.forename: L A
authors.initials: LA
authors.lastname: Lockman
mesh_terms: D000818:Animals; D001812:Blood-Brain Barrier; D001854:Bone Marrow Cells; D016026:Bone Marrow Transplantation / Q000276:immunology / Q000473:pathology*; D019070:Cell Lineage; D002465:Cell Movement; D002490:Central Nervous System / Q000166:cytology*; D005260:Female; D006086:Graft vs Host Disease / Q000473:pathology; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000628:therapy*; D008297:Male; D017628:Microglia / Q000166:cytology* / Q000201:enzymology / Q000276:immunology; D018901:Peroxisomal Disorders / Q000628:therapy*; D010587:Phagocytosis