Journal of Cellular and Molecular Medicine 2019-Sep
Mannose 6-phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease.
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概要
In the search of a better enzyme therapy in Pompe disease, the conjugation of mannose 6-phosphonates to the recombinant enzyme appeared as an enhancer of its efficacy. Here, we demonstrated that the increased efficacy of the conjugated enzyme is partly due to a higher intracellular maturation because of its insensitiveness to acid phosphatases during the routing to lysosomes.