Interim safety and efficacy of gene therapy for RLBP1-associated retinal dystrophy: a phase 1/2 trial
RLBP1相关视网膜营养不良基因疗法的中期安全性和有效性:一项1/2期试验
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-024-51575-4
Kvanta, Anders; Rangaswamy, Nalini; Holopigian, Karen; Watters, Christine; Jennings, Nicki; Liew, Melissa S H; Bigelow, Chad; Grosskreutz, Cynthia; Burstedt, Marie; Venkataraman, Abinaya; Westman, Sofie; Geirsdottir, Asbjörg; Stasi, Kalliopi; André, Helder