Carbohydrate-remodelled acid α-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

To enhance the delivery of rhGAA (recombinant GAA, where GAA stands for acid α-glucosidase) to the affected muscles in Pompe disease, the carbohydrate moieties on the enzyme were remodelled to exhibit a high affinity ligand for the CI-MPR (cation-independent M6P receptor, where M6P stands for mannos...

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Autori principali: Zhu, Yunxiang, Li, Xuemei, Mcvie-Wylie, Alison, Jiang, Canwen, Thurberg, Beth L., Raben, Nina, Mattaliano, Robert J., Cheng, Seng H.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Portland Press Ltd. 2005
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC1180711/
https://ncbi.nlm.nih.gov/pubmed/15839836
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1042/BJ20050364
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