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

Godefroy, Anastasia; Daurat, Morgane; Da Silva, Afitz; Basile, Ilaria; El Cheikh, Khaled; Caillaud, Catherine; Sacconi, Sabrina; Schoser, Benedikt; Charbonne, Henry-Vincent; Gary-Bobo, Magali; Morere, Alain; Garcia, Marcel und Maynadier, Marie (2019): Mannose 6-phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease. In: Journal of Cellular and Molecular Medicine, Bd. 23, Nr. 9: S. 6499-6503

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.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 1582-1838
• Sprache: Englisch