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Gene therapy is a promising approach for treating mucopolysaccharidoses. This is the first study of gene therapy for Morquio A (MPS IVA). Retroviral-mediated gene transfer was performed in vitro achieving N-acetylgalactosamine-6-sulfate sulfatase (GALNS) enzyme activity levels substantially higher than those observed in normal non-transduced cells, leading to the reduction of the accumulation of the specific glycosaminoglycan (GAG) keratan sulfate (KS) and chondroitin-6-sulfate (C6S).
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This page is a summary of: Various Cells Retrovirally Transduced withN-Acetylgalactosoamine-6-Sulfate Sulfatase Correct Morquio Skin FibroblastsIn Vitro, Human Gene Therapy, November 2001, Mary Ann Liebert Inc,
DOI: 10.1089/104303401753204571.
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