α-Galactosidase A Expressed in the Salivary Glands Partially Corrects Organ Biochemical Deficits in the Fabry Mouse Through Endocrine Trafficking

Fabry disease is caused by an X-linked deficiency of the lysosomal enzyme α-galactosidase A (GLA) and has been treated successfully with enzyme replacement therapy (ERT). Gene therapy has been proposed as an alternative to ERT due to the presumed advantages of continuous, endogenous production of th...

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Detaylı Bibliyografya
Asıl Yazarlar: Passineau, Michael J., Fahrenholz, Timothy, Machen, Laurie, Zourelias, Lee, Nega, Katherine, Paul, Rachel, MacDougall, Mary J., Mamaeva, Olga, Steet, Richard, Barnes, Jarrod, Kingston, H.M., Benza, Raymond L.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2011
Konular:
Research Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3057212/
https://ncbi.nlm.nih.gov/pubmed/20858137
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2010.069
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