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...

Descripció completa

Guardat en:
Dades bibliogràfiques
Autors principals: 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
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2011
Matèries:
Research Articles
Accés en línia: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
Etiquetes: Afegir etiqueta
Sense etiquetes, Sigues el primer a etiquetar aquest registre!

Ítems similars