Collection of a mobilized peripheral blood apheresis product from a patient with mucopolysaccharidosis type VII and subsequent CD34+ cell isolation.


The effectiveness of bone marrow transplantation for lysosomal storage diseases like mucopolysaccharidosis type VII (MPSVII) suggests that a gene therapy strategy targeting autologous hematopoietic progenitor cells could be successful. Given the severe systemic manifestations of MPSVII including storage disease in the bone and bone marrow, it was unclear… (More)


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