Bone marrow transplantation and gene therapy for lysosomal storage diseases

Allogeneic bone marrow transplantation with marrow from HLA-identical donors has been used with increasing frequency to treat patients with lysosomal storage diseases. The effects of bone marrow transplantation largely depend on the type and stage of the disease. Generally, 'visceral symptoms' can be largely improved by transplantation, but skeletal lesions are relatively unaffected by transplantation. The effect on neurological symptoms varies.