Lentivirus-mediated gene therapy for Fabry disease

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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Main Authors: 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
Format: Article
Language:English
Published: Nature Publishing Group 2021-02-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-021-21371-5
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spelling doaj-e1df374ec9104eca99e1b0892655fcfa2021-03-11T11:33:22ZengNature Publishing GroupNature Communications2041-17232021-02-011211910.1038/s41467-021-21371-5Lentivirus-mediated gene therapy for Fabry diseaseAneal Khan0Dwayne L. Barber1Ju Huang2C. Anthony Rupar3Jack W. Rip4Christiane Auray-Blais5Michel Boutin6Pamela O’Hoski7Kristy Gargulak8William M. McKillop9Graeme Fraser10Syed Wasim11Kaye LeMoine12Shelly Jelinski13Ahsan Chaudhry14Nicole Prokopishyn15Chantal F. Morel16Stephen Couban17Peter R. Duggan18Daniel H. Fowler19Armand Keating20Michael L. West21Ronan Foley22Jeffrey A. Medin23Department of Medical Genetics, Metabolics and Pediatrics, Alberta Children’s Hospital, Cumming School of Medicine, Research Institute, University of CalgaryUniversity Health NetworkUniversity Health NetworkDepartment of Pathology and Laboratory Medicine, Western UniversityDepartment of Pathology and Laboratory Medicine, Western UniversityDivision of Medical Genetics, Department of Pediatrics, CIUSSS de l’Estrie-CHUS Hospital Fleurimont, Université de SherbrookeDivision of Medical Genetics, Department of Pediatrics, CIUSSS de l’Estrie-CHUS Hospital Fleurimont, Université de SherbrookeDepartment of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer CentreDepartment of Pediatrics, Medical College of WisconsinDepartment of Pediatrics, Medical College of WisconsinDepartment of Oncology, McMaster University and Juravinski Hospital and Cancer CentreCancer Clinical Research Unit, Princess Margaret Cancer CentreNova Scotia Health Authority, QEII Health Sciences Centre, Canadian Fabry Disease Initiative, Nova Scotia Fabry Disease ProgramAlberta Children’s Hospital and Foothills Medical CentreDepartments of Oncology and Medicine, Alberta Blood and Marrow Transplant Program, University of CalgaryDepartment of Pathology and Laboratory Medicine, Cumming School of Medicine, University of CalgaryFred A. Litwin Family Centre in Genetic Medicine, Department of Medicine, University Health NetworkDivision of Hematology, Department of Medicine, Dalhousie UniversityCumming School of Medicine, University of CalgaryRapa TherapeuticsUniversity Health NetworkDivision of Nephrology, Department of Medicine, Dalhousie UniversityDepartment of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer CentreUniversity Health NetworkTreatments 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.https://doi.org/10.1038/s41467-021-21371-5
collection DOAJ
language English
format Article
sources DOAJ
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
spellingShingle 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
Nature Communications
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 Publishing Group
series Nature Communications
issn 2041-1723
publishDate 2021-02-01
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.
url https://doi.org/10.1038/s41467-021-21371-5
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