Gene therapy for hemophilia: an imperative to succeed
C Hough, D Lillicrap - Journal of Thrombosis and Haemostasis, 2005 - jthjournal.org
Significant advances have been made in the development of gene therapy for hemophilia in
the past decade (Fig. 1), but this progress has been accompanied by additional challenges.
Consequently, it is not altogether surprising that the biotechnology and pharmaceutical
industries have, at least for the present, withdrawn from clinical trials of gene therapy for this
disorder. The high cost associated with conducting such trials and the relatively small size of
the hemophilia patient population are no doubt factors that have, in part, influenced this …
the past decade (Fig. 1), but this progress has been accompanied by additional challenges.
Consequently, it is not altogether surprising that the biotechnology and pharmaceutical
industries have, at least for the present, withdrawn from clinical trials of gene therapy for this
disorder. The high cost associated with conducting such trials and the relatively small size of
the hemophilia patient population are no doubt factors that have, in part, influenced this …