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

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 becaus...

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Detalles Bibliográficos
Autores principales: Godefroy, Anastasia, Daurat, Morgane, Da Silva, Afitz, Basile, Ilaria, El Cheikh, Khaled, Caillaud, Catherine, Sacconi, Sabrina, Schoser, Benedikt, Charbonné, Henry‐Vincent, Gary‐Bobo, Magali, Morère, Alain, Garcia, Marcel, Maynadier, Marie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2019
Materias:
Short Communications
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6714136/
https://www.ncbi.nlm.nih.gov/pubmed/31293082
http://dx.doi.org/10.1111/jcmm.14516
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author Godefroy, Anastasia
Daurat, Morgane
Da Silva, Afitz
Basile, Ilaria
El Cheikh, Khaled
Caillaud, Catherine
Sacconi, Sabrina
Schoser, Benedikt
Charbonné, Henry‐Vincent
Gary‐Bobo, Magali
Morère, Alain
Garcia, Marcel
Maynadier, Marie
author_facet Godefroy, Anastasia
Daurat, Morgane
Da Silva, Afitz
Basile, Ilaria
El Cheikh, Khaled
Caillaud, Catherine
Sacconi, Sabrina
Schoser, Benedikt
Charbonné, Henry‐Vincent
Gary‐Bobo, Magali
Morère, Alain
Garcia, Marcel
Maynadier, Marie
author_sort Godefroy, Anastasia
collection PubMed
description 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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spelling pubmed-67141362019-09-05 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 Charbonné, Henry‐Vincent Gary‐Bobo, Magali Morère, Alain Garcia, Marcel Maynadier, Marie J Cell Mol Med Short Communications 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. John Wiley and Sons Inc. 2019-07-10 2019-09 /pmc/articles/PMC6714136/ /pubmed/31293082 http://dx.doi.org/10.1111/jcmm.14516 Text en © 2019 The Authors. Journal of Cellular and Molecular Medicine published by John Wiley & Sons Ltd and Foundation for Cellular and Molecular Medicine. This is an open access article under the terms of the http://creativecommons.org/licenses/by/4.0/ License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
spellingShingle Short Communications
Godefroy, Anastasia
Daurat, Morgane
Da Silva, Afitz
Basile, Ilaria
El Cheikh, Khaled
Caillaud, Catherine
Sacconi, Sabrina
Schoser, Benedikt
Charbonné, Henry‐Vincent
Gary‐Bobo, Magali
Morère, Alain
Garcia, Marcel
Maynadier, Marie
Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title_full Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title_fullStr Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title_full_unstemmed Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title_short Mannose 6‐phosphonate labelling: A key for processing the therapeutic enzyme in Pompe disease
title_sort mannose 6‐phosphonate labelling: a key for processing the therapeutic enzyme in pompe disease
topic Short Communications
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6714136/
https://www.ncbi.nlm.nih.gov/pubmed/31293082
http://dx.doi.org/10.1111/jcmm.14516
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