Journal article
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). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells. Gene-augmented HSPCs can …
Authors
Khan A; Barber DL; McKillop WM; Rupar CA; Auray‐Blais C; Fraser G; Fowler DH; Berger A; Foley R; Keating A
Journal
Clinical and Translational Medicine, Vol. 15, No. 1,
Publisher
Wiley
Publication Date
1 2025
DOI
10.1002/ctm2.70073
ISSN
2001-1326