AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial
AAV9基因疗法治疗GM1神经节苷脂沉积症II型:一项1/2期临床试验
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doi:10.1101/2025.07.28.25332074
Lewis, Connor J; D'Souza, Precilla; Johnston, Jean M; Acosta, Maria T; Farmer, Cristan; Baker, Eva H; Crowell, Anna; Mojica, Yoliann; Rahman, Sumaiya; Joseph, Lisa; Hartman, Adam; Vézina, Gilbert; Quezado, Zenaide; Yousef, Muhammad H; Luckett, Amelia; Vardar, Zeynep; Shazeeb, Mohammad Salman; Corti, Manuela; Blackwood, Meghan; Coleman, Kirsten; Thurm, Audrey; De Boever, Erika; Gahl, William A; Byrne, Barry J; Flotte, Terence R; Jiang, Xuntian; Gross, Amanda L; Keeler, Allison M; Gray-Edwards, Heather; Martin, Douglas R; Sena-Esteves, Miguel; Tifft, Cynthia J