Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep
酶替代疗法治疗小鼠和绵羊CLN1疾病的跨物种疗效
期刊:Journal of Clinical Investigation
影响因子:13.6
doi:10.1172/JCI163107
Nelvagal, Hemanth R; Eaton, Samantha L; Wang, Sophie H; Eultgen, Elizabeth M; Takahashi, Keigo; Le, Steven Q; Nesbitt, Rachel; Dearborn, Joshua T; Siano, Nicholas; Puhl, Ana C; Dickson, Patricia I; Thompson, Gerard; Murdoch, Fraser; Brennan, Paul M; Gray, Mark; Greenhalgh, Stephen N; Tennant, Peter; Gregson, Rachael; Clutton, Eddie; Nixon, James; Proudfoot, Chris; Guido, Stefano; Lillico, Simon G; Whitelaw, C Bruce A; Lu, Jui-Yun; Hofmann, Sandra L; Ekins, Sean; Sands, Mark S; Wishart, Thomas M; Cooper, Jonathan D