Lentivirus-mediated gene therapy for Fabry disease

Enzyme and chaperone therapies are used to treat Fabry disease. Such treatments are expensive and require intrusive biweekly infusions; they are also not particularly efficacious. In this pilot, single-arm study (NCT02800070), five adult males with Type 1 (classical) phenotype Fabry disease were inf...

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Detalles Bibliográficos
Autores principales: Khan, Aneal, Barber, Dwayne L., Huang, Ju, Rupar, C. Anthony, Rip, Jack W., Auray-Blais, Christiane, Boutin, Michel, O’Hoski, Pamela, Gargulak, Kristy, McKillop, William M., Fraser, Graeme, Wasim, Syed, LeMoine, Kaye, Jelinski, Shelly, Chaudhry, Ahsan, Prokopishyn, Nicole, Morel, Chantal F., Couban, Stephen, Duggan, Peter R., Fowler, Daniel H., Keating, Armand, West, Michael L., Foley, Ronan, Medin, Jeffrey A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7907075/
https://www.ncbi.nlm.nih.gov/pubmed/33633114
http://dx.doi.org/10.1038/s41467-021-21371-5

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