Nature Communications (Feb 2021)
Lentivirus-mediated gene therapy for Fabry disease
- 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
Affiliations
- Aneal Khan
- Department of Medical Genetics, Metabolics and Pediatrics, Alberta Children’s Hospital, Cumming School of Medicine, Research Institute, University of Calgary
- Dwayne L. Barber
- University Health Network
- Ju Huang
- University Health Network
- C. Anthony Rupar
- Department of Pathology and Laboratory Medicine, Western University
- Jack W. Rip
- Department of Pathology and Laboratory Medicine, Western University
- Christiane Auray-Blais
- Division of Medical Genetics, Department of Pediatrics, CIUSSS de l’Estrie-CHUS Hospital Fleurimont, Université de Sherbrooke
- Michel Boutin
- Division of Medical Genetics, Department of Pediatrics, CIUSSS de l’Estrie-CHUS Hospital Fleurimont, Université de Sherbrooke
- Pamela O’Hoski
- Department of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer Centre
- Kristy Gargulak
- Department of Pediatrics, Medical College of Wisconsin
- William M. McKillop
- Department of Pediatrics, Medical College of Wisconsin
- Graeme Fraser
- Department of Oncology, McMaster University and Juravinski Hospital and Cancer Centre
- Syed Wasim
- Cancer Clinical Research Unit, Princess Margaret Cancer Centre
- Kaye LeMoine
- Nova Scotia Health Authority, QEII Health Sciences Centre, Canadian Fabry Disease Initiative, Nova Scotia Fabry Disease Program
- Shelly Jelinski
- Alberta Children’s Hospital and Foothills Medical Centre
- Ahsan Chaudhry
- Departments of Oncology and Medicine, Alberta Blood and Marrow Transplant Program, University of Calgary
- Nicole Prokopishyn
- Department of Pathology and Laboratory Medicine, Cumming School of Medicine, University of Calgary
- Chantal F. Morel
- Fred A. Litwin Family Centre in Genetic Medicine, Department of Medicine, University Health Network
- Stephen Couban
- Division of Hematology, Department of Medicine, Dalhousie University
- Peter R. Duggan
- Cumming School of Medicine, University of Calgary
- Daniel H. Fowler
- Rapa Therapeutics
- Armand Keating
- University Health Network
- Michael L. West
- Division of Nephrology, Department of Medicine, Dalhousie University
- Ronan Foley
- Department of Pathology and Molecular Medicine, McMaster University and Juravinski Hospital and Cancer Centre
- Jeffrey A. Medin
- University Health Network
- DOI
- https://doi.org/10.1038/s41467-021-21371-5
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 9
Abstract
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.
