Proof-of-concept for multiple AON delivery by a single U7snRNA vector to restore splicing defects in ABCA4
利用单个 U7snRNA 载体递送多个 AON 以恢复 ABCA4 剪接缺陷的概念验证
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2024.01.019
Suárez-Herrera, Nuria; Riswick, Iris B; Vázquez-Domínguez, Irene; Duijkers, Lonneke; Karjosukarso, Dyah W; Piccolo, Davide; Bauwens, Miriam; De Baere, Elfride; Cheetham, Michael E; Garanto, Alejandro; Collin, Rob W J