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Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease

Improving the delivery of therapeutics to disease-affected tissues can increase their efficacy and safety. Here, we show that chemical conjugation of a synthetic oligosaccharide harboring mannose 6-phosphate (M6P) residues onto recombinant human acid α-glucosidase (rhGAA) via oxime chemistry signifi...

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書誌詳細
主要な著者: Zhu, Yunxiang, Jiang, Ji-Lei, Gumlaw, Nathan K, Zhang, Jinhua, Bercury, Scott D, Ziegler, Robin J, Lee, Karen, Kudo, Mariko, Canfield, William M, Edmunds, Timothy, Jiang, Canwen, Mattaliano, Robert J, Cheng, Seng H
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2009
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC2835178/
https://ncbi.nlm.nih.gov/pubmed/19277015
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.37
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