Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5
SAR422459 (EIAV-ABCA4) 基因疗法治疗 ABCA4 相关 Stargardt 病患者的三年安全性结果：一项开放标签剂量递增 I/IIa 期临床试验，队列 1-5
期刊:American Journal of Ophthalmology
影响因子:4.2
doi:10.1016/j.ajo.2022.02.013
Parker, Maria A; Erker, Laura R; Audo, Isabelle; Choi, Dongseok; Mohand-Said, Saddek; Sestakauskas, Kastytis; Benoit, Patrick; Appelqvist, Terence; Krahmer, Melissa; Ségaut-Prévost, Caroline; Lujan, Brandon J; Faridi, Ambar; Chegarnov, Elvira N; Steinkamp, Peter N; Ku, Cristy; da Palma, Mariana Matioli; Barale, Pierre-Olivier; Ayelo-Scheer, Sarah; Lauer, Andreas; Stout, Tim; Wilson, David J; Weleber, Richard G; Pennesi, Mark E; Sahel, José Alain; Yang, Paul
