Bone Marrow Transplant
Hematopoietic cell transplantation augmented by enzyme replacement therapy in Hurler syndrome
Published in Gene Therapy Weekly, August 31st, 2006
"MPS 1H is the severe form of mucopolysaccharidosis type 1 (MPS 1). HCT is the treatment of choice, but carries a high incidence of graft failure and morbidity.
"The use of ERT might improve the clinical signs and symptoms before HCT, resulting in less transplantation-related complications. Moreover, clearance of glycosaminoglycans (GAG's) from the bone marrow might improve engraftment," wrote J. Cox-Brinkman and colleagues, University of Utrecht.
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Source: Gene Therapy Weekly (2006-08-31)
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