Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial

Lentivirus‐mediated gene therapy for Fabry disease: 5‐year End‐of‐Study results from the Canadian FACTs trial

Abstract Background Fabry disease is an X‐linked lysosomal storage disorder due to a deficiency of α‐galactosidase A (α‐gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α‐gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs)....

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Main Authors: Aneal Khan, Dwayne L. Barber, William M. McKillop, C. Anthony Rupar, Christiane Auray‐Blais, Graeme Fraser, Daniel H. Fowler, Alexandra Berger, Ronan Foley, Armand Keating, Michael L. West, Jeffrey A. Medin
Format: Article
Language:English
Published: Wiley 2025-01-01
Series:Clinical and Translational Medicine
Subjects:
clinical trial results
haematopoietic stem cells
lysosomal storage disorders
melphalan conditioning
progenitor cells
Online Access:https://doi.org/10.1002/ctm2.70073
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https://doi.org/10.1002/ctm2.70073

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