Single Cell Analysis and Selection of Living Retrovirus Vector-corrected Mucopolysaccharidosis VII Cells Using a Fluorescence-activated Cell Sorting-based Assay for Mammalian β-Glucuronidase Enzymatic Activity

Mutations in the acid β-glucuronidase gene lead to systemic accumulation of undegraded glycosaminoglycans in lysosomes and ultimately to clinical manifestations of mucopolysaccharidosis VII (Sly disease). Gene transfer by retrovirus vectors into murine mucopolysaccharidosis VII hematopoietic stem ce...

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