Perspectives on gene therapy for lysosomal storage diseases that affect hematopoiesis.

Successful gene therapy for lysosomal storage diseases that involve the bone marrow and hematopoiesis is hindered by the hostile microenvironments created by these diseases and the irreversibility of the pathologic manifestations. Of the 40 lysosomal storage diseases, Gaucher disease type I, Niemann-Pick B, and mucopolysaccharidosis type I have hematopoietic involvement, are treatable by enzyme therapy, and respond to bone marrow transplantation. These storage diseases provide the basis for continued development of gene therapy.