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Fabry Disease: Preclinical Studies Demonstrate the Effectiveness of α-Galactosidase A Replacement in Enzyme-Deficient Mice

Preclinical studies of enzyme-replacement therapy for Fabry disease (deficient α-galactosidase A [α-Gal A] activity) were performed in α-Gal A–deficient mice. The pharmacokinetics and biodistributions were determined for four recombinant human α-Gal A glycoforms, which differed in sialic acid and ma...

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Bibliografiske detaljer
Main Authors: Ioannou, Yiannis A., Zeidner, Ken M., Gordon, Ronald E., Desnick, Robert J.
Format: Artigo
Sprog:Inglês
Udgivet: The American Society of Human Genetics 2001
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1234907/
https://ncbi.nlm.nih.gov/pubmed/11115376
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