Open AccessMannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
Author(s) -
Godefroy Anastasia,
Daurat Morgane,
Da Silva Afitz,
Basile Ilaria,
El Cheikh Khaled,
Caillaud Catherine,
Sacconi Sabrina,
Schoser Benedikt,
Charbonné HenryVincent,
GaryBobo Magali,
Morère Alain,
Garcia Marcel,
Maynadier Marie
Publication year - 2019
Publication title -
journal of cellular and molecular medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.44
H-Index - 130
eISSN - 1582-4934
pISSN - 1582-1838
DOI - 10.1111/jcmm.14516
Subject(s) - mannose , enzyme , enzyme replacement therapy , biochemistry , labelling , mannose 6 phosphate receptor , recombinant dna , intracellular , phosphatase , chemistry , enhancer , disease , lysosome , medicine , gene expression , gene
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.