Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
慢病毒基因疗法治疗脑型肾上腺脑白质营养不良
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa2400442
Eichler, Florian; Duncan, Christine N; Musolino, Patricia L; Lund, Troy C; Gupta, Ashish O; De Oliveira, Satiro; Thrasher, Adrian J; Aubourg, Patrick; Kühl, Jörn-Sven; Loes, Daniel J; Amartino, Hernan; Smith, Nicholas; Folloni Fernandes, Juliana; Sevin, Caroline; Sankar, Raman; Hussain, Shaun A; Gissen, Paul; Dalle, Jean-Hugues; Platzbecker, Uwe; Downey, Gerald F; McNeil, Elizabeth; Demopoulos, Laura; Dietz, Andrew C; Thakar, Himal L; Orchard, Paul J; Williams, David A