Orkambi® and amplifier co-therapy improves function from a rare CFTR mutation in gene-edited cells and patient tissue
Orkambi® 和放大器联合疗法可改善基因编辑细胞和患者组织中罕见 CFTR 突变的功能
期刊:EMBO Molecular Medicine
影响因子:9
doi:10.15252/emmm.201607137
Steven V Molinski, Saumel Ahmadi, Wan Ip, Hong Ouyang, Adriana Villella, John P Miller, Po-Shun Lee, Kethika Kulleperuma, Kai Du, Michelle Di Paola, Paul Dw Eckford, Onofrio Laselva, Ling Jun Huan, Leigh Wellhauser, Ellen Li, Peter N Ray, Régis Pomès, Theo J Moraes, Tanja Gonska, Felix Ratjen, Chris
