In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis
在囊性纤维化雪貂模型中,子宫内和出生后给予VX-770可挽救多器官疾病
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.aau7531
Sun, Xingshen; Yi, Yaling; Yan, Ziying; Rosen, Bradley H; Liang, Bo; Winter, Michael C; Evans, T Idil Apak; Rotti, Pavana G; Yang, Yu; Gray, Jaimie S; Park, Soo Yeun; Zhou, Weihong; Zhang, Yulong; Moll, Shashanna R; Woody, Lisa; Tran, Dao M; Jiang, Licong; Vonk, Annelotte M; Beekman, Jeffrey M; Negulescu, Paul; Van Goor, Fred; Fiorino, Dennis F; Gibson-Corley, Katherine N; Engelhardt, John F