Leukodystrophy and Bone Marrow Transplantation: Role of Mixed Hematopoietic Chimerism

Bone Marrow Transplantation (BMT) is currently the most physiologic treatment for some types of leukodystrophies. In enzyme deficiency states, replacement of defective genes with cells carrying “normal” copies of these genes offers a natural form of gene therapy. This review will cover the various disease states which may be treated using bone marrow… CONTINUE READING