Journal article
Lentivirus-mediated gene therapy for Fabry disease
Abstract
Enzyme and chaperone therapies are used to treat Fabry disease. Such treatments are expensive and require intrusive biweekly infusions; they are also not particularly efficacious. In this pilot, single-arm study (NCT02800070), five adult males with Type 1 (classical) phenotype Fabry disease were infused with autologous lentivirus-transduced, CD34+-selected, hematopoietic stem/progenitor cells engineered to express alpha-galactosidase A (α-gal …
Authors
Khan A; Barber DL; Huang J; Rupar CA; Rip JW; Auray-Blais C; Boutin M; O’Hoski P; Gargulak K; McKillop WM
Journal
Nature Communications, Vol. 12, No. 1,
Publisher
Springer Nature
DOI
10.1038/s41467-021-21371-5
ISSN
2041-1723