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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Publicat a:Nat Commun
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group UK 2021
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7907075/
https://ncbi.nlm.nih.gov/pubmed/33633114
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-021-21371-5
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