Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
The New England Journal Of Medicine
George, Lindsey A LA; Sullivan, Spencer K SK; Giermasz, Adam A; Rasko, John E J JEJ; Samelson-Jones, Benjamin J BJ; Ducore, Jonathan J; Cuker, Adam A; Sullivan, Lisa M LM; Majumdar, Suvankar S; Teitel, Jerome J; McGuinn, Catherine E CE; Ragni, Margaret V MV; Luk, Alvin Y AY; Hui, Daniel D; Wright, J Fraser JF; Chen, Yifeng Y; Liu, Yun Y; Wachtel, Katie K; Winters, Angela A; Tiefenbacher, Stefan S; Arruda, Valder R VR; van der Loo, Johannes C M JCM; Zelenaia, Olga O; Takefman, Daniel D; Carr, Marcus E ME; Couto, Linda B LB; Anguela, Xavier M XM; High, Katherine A KA