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.

• Authors: 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: Journal of cellular and molecular medicine (J Cell Mol Med) Vol. 23 Issue 9 Pg. 6499-6503 (09 2019) ISSN: 1582-4934 [Electronic] England
• PMID: 31293082 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2019 The Authors. Journal of Cellular and Molecular Medicine published by John Wiley & Sons Ltd and Foundation for Cellular and Molecular Medicine.

Chemical References

• Organophosphonates
• Mannose

Topics

• Adult
• Animals
• Cells, Cultured
• Disease Models, Animal
• Glycogen Storage Disease Type II (metabolism)
• Humans
• Lysosomes (metabolism)
• Mannose (metabolism)
• Mice
• Muscle, Skeletal (metabolism)
• Myoblasts (metabolism)
• Organophosphonates (metabolism)