The mucopolysaccharidoses (MPS) are lysosomal storage diseases which are characterized by multiple organ involvement, progressive deterioration and premature death. No definitive safe therapy is available, although numerous therapies, such as plasma exchange, lymphocyte infusions and transplantation of fibroblasts have been attempted with minimal success. Bone marrow transplantation has been attempted, but it has significant mortality. The rationale for all these procedures is the ability of deficient fibroblasts to take up lysosomal enzymes supplied by the donor's plasma or cells. Children with MPS will undergo transplantation of human amniotic membranes to evaluate this procedure as a possible treatment in the MPS disorders. Clinical use of amniotic membranes, such as biological dressings, have shown this tissue to have low immunogenicity. Amniotic epithelial cells produce lysosomal enzymes and can correct in vitro the enzymatic defect in Hurler syndrome fibroblasts. A second transplant of amniotic membrane and biopsy of initial transplantation site will be performed to examine the viability of transplanted material and to determine if the response can be elicited repeatedly. This clinical trial will be evaluated by frequent clinical and biochemical monitoring.
