Sustained Clinical Benefit of AAV Gene Therapy in Severe Hemophilia B
AAV基因疗法在重度B型血友病中持续发挥临床疗效
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2414783
Reiss, Ulrike M; Davidoff, Andrew M; Tuddenham, Edward G D; Chowdary, Pratima; McIntosh, Jenny; Muczynski, Vincent; Journou, Malo; Simini, Giulia; Ireland, Lydia; Mohamed, Saira; Riddell, Anne; Pie, Arnulfo J; Hall, Andrew; Quaglia, Alberto; Mangles, Sarah; Mahlangu, Johnny; Haley, Kristina; Recht, Michael; Shen, Yu-Min; Halka, Kathleen G; Fortner, Gail; Morton, Christopher L; Gu, Zhengming; Hayden, Randall T; Neufeld, Ellis J; Okhomina, Victoria I; Kang, Guolian; Nathwani, Amit C