Pm. Hoogerbrugge et D. Valerio, BONE-MARROW TRANSPLANTATION AND GENE-THERAPY FOR LYSOSOMAL STORAGE DISEASES, Bone marrow transplantation, 21, 1998, pp. 34-36
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, 'visc
eral symptoms' can be largely improved by transplantation, but skeleta
l lesions are relatively unaffected by transplantation. The effect on
neurological symptoms varies.