Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice
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DOI: 10.1016/j.ymgmr.2019.100510
Abstract: Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder (LSD) characterized by severe central nervous system (CNS) degeneration. The disease is caused by mutations in the SGSH gene coding for the lysosomal enzyme sulfamidase.… read more here.
Keywords: sulfamidase; heparan sulfate; pathology; mps iiia ... See more keywords