Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
玻璃体内注射反义寡核苷酸 sepofarsen 治疗 Leber 先天性黑蒙 10 型:一项 1b/2 期试验
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-022-01755-w
Russell, Stephen R; Drack, Arlene V; Cideciyan, Artur V; Jacobson, Samuel G; Leroy, Bart P; Van Cauwenbergh, Caroline; Ho, Allen C; Dumitrescu, Alina V; Han, Ian C; Martin, Mitchell; Pfeifer, Wanda L; Sohn, Elliott H; Walshire, Jean; Garafalo, Alexandra V; Krishnan, Arun K; Powers, Christian A; Sumaroka, Alexander; Roman, Alejandro J; Vanhonsebrouck, Eva; Jones, Eltanara; Nerinckx, Fanny; De Zaeytijd, Julie; Collin, Rob W J; Hoyng, Carel; Adamson, Peter; Cheetham, Michael E; Schwartz, Michael R; den Hollander, Wilhelmina; Asmus, Friedrich; Platenburg, Gerard; Rodman, David; Girach, Aniz