Journal article
Past, present and future of haemophilia gene therapy: From vectors and transgenes to known and unknown outcomes
Abstract
Since the 1960s, the pace of innovation in haemophilia treatment has been fast and furious and occasionally with unintended consequences. As newer technologies are harnessed to better treat, and potentially cure, haemophilias A and B, an understanding of their underlying scientific principles and their benefits and risks are essential for all stakeholders. This review summarizes the starts and stops of introducing FVIII and FIX transgenes …
Authors
Pierce GF; Iorio A
Journal
Haemophilia, Vol. 24, No. S6, pp. 60–67
Publisher
Wiley
Publication Date
5 2018
DOI
10.1111/hae.13489
ISSN
1351-8216