Bone marrow transplantation therapy for metabolic disease: animal models as predictors of success and in utero approaches.

A large series of animal models of human lysosomal storage diseases has undergone bone marrow transplantation (BMT) to evaluate this approach to providing a source of normal lysosomal hydrolases for therapy. The relative success of this approach appears to be dependent on the age at transplant and the specific disease being treated. The variability in results between different diseases may be due to the observation that the amount of each specific enzyme secreted by bone marrow and bone marrow-derived cells varies, and because different diseases have different organ systems which are affected, with particular emphasis on the central nervous system pathology.