Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial
基因治疗后,莱伯西林相关性视网膜纤毛病患者视锥细胞介导的视觉功能恢复:一项 I/II 期试验的一年结果
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.06.035
Aleman, Tomas S; Uyhazi, Katherine E; Roman, Alejandro J; Weber, Mariejel L; O'Neil, Erin C; Swider, Malgorzata; Sumaroka, Alexander; Maguire, Katherine H; Aleman, Elena M; Santos, Arlene J; Kim, Rebecca J; Parchinski, Kelsey M; Billek, Andrew; Fradin, Makayla; Chung, William; Margaritis, Paris; Sun, Junwei; Scoles, Drew H; Wu, Vivian; Garafalo, Alexandra V; Jayagopal, Ashwath; Yerxa, Ben; Tuller, Sarah; Maguire, Albert M; Bennett, Jean; Cideciyan, Artur V