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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

Fabry disease, a lysosomal storage disorder resulting from the deficient activity of α-galactosidase A (α-Gal A), is characterized by cardiac, renal, and/or cerebrovascular disease due to progressive accumulation of the enzyme’s substrates, globotriaosylceramide (Gb3) and globotriaosylsphingosine (L...

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Detalles Bibliográficos
Autores principales:	Pagant, Silvere, Huston, Marshall W., Moreira, Luciana, Gan, Lin, St Martin, Susan, Sproul, Scott, Holmes, Michael C., Meyer, Kathleen, Wechsler, Thomas, Desnick, Robert J., Yasuda, Makiko
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8572137/ https://www.ncbi.nlm.nih.gov/pubmed/33775910 http://dx.doi.org/10.1016/j.ymthe.2021.03.018

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