Therapeutic approaches to lysosomal storage diseases

Abstract

Nascent therapies for the lysosomal storage diseases have begun. The replacement enzyme therapy for Gaucher's disease now includes a recombinant form, and effective dosing schedules are being developed. Bone marrow transplantation appears to be a very successful treatment for nonneuronopathic Gaucher's disease and halts the progression of other lysosomal storage disorders. Following the success of bone marrow transplantation, gene therapy trials using transduced human hematopoietic cells are beginning in Gaucher's disease, which should lead to autologous bone marrow transplantation using genetically engineered cells. Experimental studies hold promise for neurologic treatment in the lysosomal storage diseases using transplanted recombinant cells and neural progenitor cells.