Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

A. George, Lindsey; K. Sullivan, Spencer; Giermasz, Adam; E.J. Rasko, John; J. Samelson‐Jones, Benjamin; M. Ducore, Jonathan; Cuker, Adam; Sullivan, Lisa; Majumdar, Suvankar; Teitel, Jerome; McGuinn, Catherine; V. Ragni, Margaret; Luk, Alvin; J. Hui, Daniel; Fraser Wright, J.; Chen, Yifeng; Liu, Yun; Wachtel, Katie; Winters, Angela; Tiefenbacher, Stefan; R. Arruda, Valder; C.M. van der Loo, Johannes; Zelenaia, Olga; M. Takefman, Daniel; E. Carr, Marcus; B. Couto, Linda; M. Anguela, Xavier; A. High, Katherine

doi:10.1056/nejmoa1708538

Public

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Published in New England Journal of Medicine • Dec 6, 2017

Authors:

Lindsey A. George

Spencer K. Sullivan

Adam Giermasz

Abstract

BACKGROUND: The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia. METHODS: vector genomes per kilogram of body weight ...

Subject

Medicine

Factor IX

Clotting factor

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!