Mannose 6-phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease

Abstract

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.

Keywords: acid phosphatases; acid α-glucosidase; enzyme replacement therapy; intracellular processing; lysosomal storage disease; mannose 6-phosphate receptor.