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

Anastasia Godefroy,Morgane Daurat,Afitz Da Silva,Ilaria Basile,Khaled El Cheikh,Catherine Caillaud,Sabrina Sacconi,Benedikt Schoser, Henry-Vincent Charbonné,Magali Gary-Bobo,Alain Morère,Marcel Garcia,Marie Maynadier

JOURNAL OF CELLULAR AND MOLECULAR MEDICINE（2019）

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摘要

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.

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关键词

acid phosphatases,acid alpha-glucosidase,enzyme replacement therapy,intracellular processing,lysosomal storage disease,mannose 6-phosphate receptor

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