Journal of Cellular and Molecular Medicine | 2019
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.