Lentivirus-mediated gene therapy for Fabry disease

Treatments for Fabry disease, an inherited lysosomal disorder caused by the deficiency of the enzyme alpha-galactosidase A, are not fully efficacious. Here the authors report a single-arm phase I trial of gene therapy with autologous, lentivirus-transduced, hematopoietic cells that express alpha-gal...

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Autores principales:	Aneal Khan, Dwayne L. Barber, Ju Huang, C. Anthony Rupar, Jack W. Rip, Christiane Auray-Blais, Michel Boutin, Pamela O’Hoski, Kristy Gargulak, William M. McKillop, Graeme Fraser, Syed Wasim, Kaye LeMoine, Shelly Jelinski, Ahsan Chaudhry, Nicole Prokopishyn, Chantal F. Morel, Stephen Couban, Peter R. Duggan, Daniel H. Fowler, Armand Keating, Michael L. West, Ronan Foley, Jeffrey A. Medin
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/e1df374ec9104eca99e1b0892655fcfa
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spelling	oai:doaj.org-article:e1df374ec9104eca99e1b0892655fcfa2021-12-02T13:33:09ZLentivirus-mediated gene therapy for Fabry disease10.1038/s41467-021-21371-52041-1723https://doaj.org/article/e1df374ec9104eca99e1b0892655fcfa2021-02-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-21371-5https://doaj.org/toc/2041-1723Treatments for Fabry disease, an inherited lysosomal disorder caused by the deficiency of the enzyme alpha-galactosidase A, are not fully efficacious. Here the authors report a single-arm phase I trial of gene therapy with autologous, lentivirus-transduced, hematopoietic cells that express alpha-galactosidase A to demonstrate that this approach is safe in five patients with Fabry disease.Aneal KhanDwayne L. BarberJu HuangC. Anthony RuparJack W. RipChristiane Auray-BlaisMichel BoutinPamela O’HoskiKristy GargulakWilliam M. McKillopGraeme FraserSyed WasimKaye LeMoineShelly JelinskiAhsan ChaudhryNicole ProkopishynChantal F. MorelStephen CoubanPeter R. DugganDaniel H. FowlerArmand KeatingMichael L. WestRonan FoleyJeffrey A. MedinNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-9 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Aneal Khan Dwayne L. Barber Ju Huang C. Anthony Rupar Jack W. Rip Christiane Auray-Blais Michel Boutin Pamela O’Hoski Kristy Gargulak William M. McKillop Graeme Fraser Syed Wasim Kaye LeMoine Shelly Jelinski Ahsan Chaudhry Nicole Prokopishyn Chantal F. Morel Stephen Couban Peter R. Duggan Daniel H. Fowler Armand Keating Michael L. West Ronan Foley Jeffrey A. Medin Lentivirus-mediated gene therapy for Fabry disease
description	Treatments for Fabry disease, an inherited lysosomal disorder caused by the deficiency of the enzyme alpha-galactosidase A, are not fully efficacious. Here the authors report a single-arm phase I trial of gene therapy with autologous, lentivirus-transduced, hematopoietic cells that express alpha-galactosidase A to demonstrate that this approach is safe in five patients with Fabry disease.
format	article
author	Aneal Khan Dwayne L. Barber Ju Huang C. Anthony Rupar Jack W. Rip Christiane Auray-Blais Michel Boutin Pamela O’Hoski Kristy Gargulak William M. McKillop Graeme Fraser Syed Wasim Kaye LeMoine Shelly Jelinski Ahsan Chaudhry Nicole Prokopishyn Chantal F. Morel Stephen Couban Peter R. Duggan Daniel H. Fowler Armand Keating Michael L. West Ronan Foley Jeffrey A. Medin
author_facet	Aneal Khan Dwayne L. Barber Ju Huang C. Anthony Rupar Jack W. Rip Christiane Auray-Blais Michel Boutin Pamela O’Hoski Kristy Gargulak William M. McKillop Graeme Fraser Syed Wasim Kaye LeMoine Shelly Jelinski Ahsan Chaudhry Nicole Prokopishyn Chantal F. Morel Stephen Couban Peter R. Duggan Daniel H. Fowler Armand Keating Michael L. West Ronan Foley Jeffrey A. Medin
author_sort	Aneal Khan
title	Lentivirus-mediated gene therapy for Fabry disease
title_short	Lentivirus-mediated gene therapy for Fabry disease
title_full	Lentivirus-mediated gene therapy for Fabry disease
title_fullStr	Lentivirus-mediated gene therapy for Fabry disease
title_full_unstemmed	Lentivirus-mediated gene therapy for Fabry disease
title_sort	lentivirus-mediated gene therapy for fabry disease
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/e1df374ec9104eca99e1b0892655fcfa
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Lentivirus-mediated gene therapy for Fabry disease

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