Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2012 Jul:18 Suppl 4:13-7.
doi: 10.1111/j.1365-2516.2012.02823.x.

Gene therapy for haemophilia B

E Tuddenham  1
Affiliations

Gene therapy for haemophilia B

E Tuddenham. Haemophilia. 2012 Jul.

Abstract

AAV virus mediated transfer of factor IX to humans is safe and effective at three dose levels. Two subjects treated at highest dose level developed immune mediated transaminitis which resolved on a short course of Prednisolone. Beneficial effects in terms of continuous elevation of factor IX level above base line was seen in all subjects, continuing for over 18 months. Further study of this treatment method is warranted.

PubMed Disclaimer

LinkOut - more resources