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

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). Overexpre...

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Veröffentlicht in:	Clinical and translational medicine 2025-01, Vol.15 (1), p.n/a
Hauptverfasser:	Khan, Aneal, Barber, Dwayne L., McKillop, William M., Rupar, C. Anthony, Auray‐Blais, Christiane, Fraser, Graeme, Fowler, Daniel H., Berger, Alexandra, Foley, Ronan, Keating, Armand, West, Michael L., Medin, Jeffrey A.
Format:	Artikel
Sprache:	eng
Schlagworte:	clinical trial results Clinical trials Enzymes Gene therapy haematopoietic stem cells Leukocytes lysosomal storage disorders melphalan conditioning Patients Plasma progenitor cells
Online-Zugang:	Volltext
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