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

Authors :: Godefroy A
Daurat M
Da Silva A
Basile I
El Cheikh K
Caillaud C
Sacconi S
Schoser B
Charbonné HV
Gary-Bobo M
Morère A
Garcia M
Maynadier M
Source :: Journal of cellular and molecular medicine [J Cell Mol Med] 2019 Sep; Vol. 23 (9), pp. 6499-6503. Date of Electronic Publication: 2019 Jul 10.
Publication Year :: 2019
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.<br /> (© 2019 The Authors. Journal of Cellular and Molecular Medicine published by John Wiley & Sons Ltd and Foundation for Cellular and Molecular Medicine.)

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

Full Text Access

Tools