Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice
BMN 270 基因治疗可使小鼠和灵长类动物的 FVIII 达到治疗水平,并使嗜血病小鼠的出血正常化
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2017.12.009
Stuart Bunting, Lening Zhang, Lin Xie, Sherry Bullens, Rajeev Mahimkar, Sylvia Fong, Krystal Sandza, Danielle Harmon, Bridget Yates, Britta Handyside, Choong-Ryoul Sihn, Nicole Galicia, Laurie Tsuruda, Charles A O'Neill, Anil Bagri, Peter Colosi, Shinong Long, Gordon Vehar, Barrie Carter