Journal article
Hemophilia gene therapy: first, do no harm
Abstract
The introduction of adeno-associated virus-mediated, liver-directed gene therapy into the hemophilia treatment landscape brings not only great promise but also considerable uncertainty to a community that has a history punctuated by the devastating effects of HIV and hepatitis C virus. These infections were introduced into people with hemophilia through the innovation of factor concentrates in the 1970s and 1980s. Concentrates, heralded as a …
Authors
Valentino LA; Kaczmarek R; Pierce GF; Noone D; O'Mahony B; Page D; Rotellini D; Skinner MW
Journal
Journal of Thrombosis and Haemostasis, Vol. 21, No. 9, pp. 2354–2361
Publisher
Elsevier
Publication Date
September 2023
DOI
10.1016/j.jtha.2023.06.016
ISSN
1538-7933