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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Opis bibliograficzny
Główni autorzy: 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.
Format: Artigo
Język:Inglês
Wydane: Mary Ann Liebert, Inc. 2011
Hasła przedmiotowe:
Research Articles
Dostęp online: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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