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Optimizing human α-galactosidase for treatment of Fabry disease

Fabry disease is caused by a deficiency of α-galactosidase A (GLA) leading to the lysosomal accumulation of globotriaosylceramide (Gb3) and other glycosphingolipids. Fabry patients experience significant damage to the heart, kidney, and blood vessels that can be fatal. Here we apply directed evoluti...

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Detalles Bibliográficos
Autores principales:	Hallows, William C., Skvorak, Kristen, Agard, Nick, Kruse, Nikki, Zhang, Xiyun, Zhu, Yu, Botham, Rachel C., Chng, Chinping, Shukla, Charu, Lao, Jessica, Miller, Mathew, Sero, Antoinette, Viduya, Judy, Ismaili, Moulay Hicham Alaoui, McCluskie, Kerryn, Schiffmann, Raphael, Silverman, Adam P., Shen, Jin-Song, Huisman, Gjalt W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10036536/ https://www.ncbi.nlm.nih.gov/pubmed/36959353 http://dx.doi.org/10.1038/s41598-023-31777-4

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