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Efficient therapy for refractory Pompe disease by mannose 6-phosphate analogue grafting on acid α-glucosidase

Archive ouverte | Basile, Ilaria | CCSD

Pompe disease is a rare disorder due to deficiency of the acid a-glucosidase (GAA) treated by enzyme replacement therapy. The present authorized treatment with rhGAA, the recombinant human enzyme, provides an important benefit in ...

Design of potent mannose 6-phosphate analogues for the functionalization of lysosomal enzymes to improve the treatment of pompe disease

Archive ouverte | El Cheikh, Khaled | CCSD

Improving therapeutics delivery in enzyme replacement therapy (ERT) for lysosomal storage disorders is a challenge. Herein, we present the synthesis of novel analogues of mannose 6-phosphate (M6P), known as AMFAs and functionalize...

Cation-independent mannose 6-phosphate receptor: From roles and functions to targeted therapies

Archive ouverte | Gauthier, Corentin | CCSD

International audience. The cation-independent mannose 6-phosphate receptor (CI-M6PR) is a ubiquitous transmembrane receptor whose main intracellular role is to direct enzymes carrying mannose 6-phosphate moieties t...

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