Bone marrow transplantation for treatment of lysosomal storage diseases. Proceedings of a colloquium. May 7, 1985, Washington, D.C.

Specific Treatment for Lysosomal Storage Disorders: Enzyme Replacement Therapy, Bone Marrow Transplant and Others

Received December 15, 2003 Abstract In this article, we review specific therapies that tackle the basic biochemical defects of lysosomal storage diseases. These include bone marrow transplantation, substrate deprivation therapy, enzyme replacement therapy and enzyme enhancement therapy. We particularly update the progress of development of enzyme replacement therapy, which plays a major role in...

Bone marrow transplantation for lysosomal storage disorders

AUTOLOGOUS MARROW TRANSPLANTATION IN THE TREATMENT OF MOPP AND ABVD-RESI STANT HODGKIN\'S DISEASE

Six patients with disseminated Hodgkin's disease resistant to MOPP (mechlorethamine, vincristine, procarbazine and prednisolone), and ABVD (adriamycin, bleomycin, vinblastine and dacarbazine) chemotherapy were treated with high-dose chemotherapy and autologous marrow transplantation. The patients first underwent marrow aspiration and storage for subsequent autologous bone marrow transplant...

Behavior and therapeutic efficacy of beta-glucuronidase-positive mononuclear phagocytes in a murine model of mucopolysaccharidosis type VII.

Bone marrow transplantation (BMT) is relatively effective for the treatment of lysosomal storage diseases. To better understand the contribution of specific hematopoietic lineages to the efficacy of BMT, we transplanted beta-glucuronidase-positive mononuclear phagocytes derived from either the peritoneum or from bone marrow in vitro into syngeneic recipients with mucopolysaccharidosis type VII ...

Behavior and Therapeutic Efficacy of b-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII

Bone marrow transplantation (BMT) is relatively effective for the treatment of lysosomal storage diseases. To better understand the contribution of specific hematopoietic lineages to the efficacy of BMT, we transplanted b-glucuronidase–positive mononuclear phagocytes derived from either the peritoneum or from bone marrow in vitro into syngeneic recipients with mucopolysaccharidosis type VII (MP...