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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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| Autori principali: | , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
The American Society of Human Genetics
2001
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1234907/ https://ncbi.nlm.nih.gov/pubmed/11115376 |
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