Intrathecal Gene Therapy for Giant Axonal Neuropathy
鞘内基因疗法治疗巨轴突神经病
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2307952
Bharucha-Goebel, Diana X; Todd, Joshua J; Saade, Dimah; Norato, Gina; Jain, Minal; Lehky, Tanya; Bailey, Rachel M; Chichester, Jessica A; Calcedo, Roberto; Armao, Diane; Foley, A Reghan; Mohassel, Payam; Tesfaye, Eshetu; Carlin, Bradley P; Seremula, Beth; Waite, Melissa; Zein, Wadih M; Huryn, Laryssa A; Crawford, Thomas O; Sumner, Charlotte J; Hoke, Ahmet; Heiss, John D; Charnas, Lawrence; Hooper, Jody E; Bouldin, Thomas W; Kang, Elizabeth M; Rybin, Denis; Gray, Steven J; Bönnemann, Carsten G