Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
利用高比活性因子IX变体治疗B型血友病
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa1708538
George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam; Rasko, John E J; Samelson-Jones, Benjamin J; Ducore, Jonathan; Cuker, Adam; Sullivan, Lisa M; Majumdar, Suvankar; Teitel, Jerome; McGuinn, Catherine E; Ragni, Margaret V; Luk, Alvin Y; Hui, Daniel; Wright, J Fraser; Chen, Yifeng; Liu, Yun; Wachtel, Katie; Winters, Angela; Tiefenbacher, Stefan; Arruda, Valder R; van der Loo, Johannes C M; Zelenaia, Olga; Takefman, Daniel; Carr, Marcus E; Couto, Linda B; Anguela, Xavier M; High, Katherine A