allogeneic hematopoietic stem cell transplantation (Masonic Cancer Center)

This allogeneic hematopoietic stem cell transplantation (HSCT) therapy is a cellular therapy modality developed and investigated by the Masonic Cancer Center at the University of Minnesota for the treatment of various inherited metabolic storage diseases. The therapy involves the infusion of donor-derived hematopoietic stem cells—sourced from bone marrow, peripheral blood, or umbilical cord blood—into patients following a conditioning regimen typically consisting of busulfan, cyclophosphamide, and antithymocyte globulin. The mechanism of action relies on the successful engraftment of donor cells, which then differentiate into various lineages, including macrophages and microglia. These donor-derived cells provide a continuous source of the functional enzymes or proteins that are deficient in the patient (such as arylsulfatase A, galactosylceramidase, or glucocerebrosidase). Through a process known as metabolic cross-correction, these functional enzymes are secreted by donor cells and taken up by the patient's own enzyme-deficient cells, thereby halting or slowing the progression of systemic and neurological symptoms associated with lysosomal and peroxisomal storage disorders.

Other names

hematopoietic stem cells-Masonic Cancer Center, University of Minnesota-adrenoleukodystrophy-metachromatic leukodystrophy-globoid cell leukodystrophy-Gaucher's disease-fucosidosis-Wolman disease-Niemann-Pick disease-Batten disease-GM1 gangliosidosis-Tay-Sachs disease-Sandhoff diseaseStem Cell TransplantBone Marrow TransplantationHCTHematopoietic Cell TransplantationUmbilical Cord Blood Transplantation