Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
利用单个AAV载体进行基因敲低和替换，实现不依赖于突变的视紫红质基因治疗
期刊:Proceedings of the National Academy of Sciences of the United States of America
影响因子:9.1
doi:10.1073/pnas.1805055115
Cideciyan, Artur V; Sudharsan, Raghavi; Dufour, Valérie L; Massengill, Michael T; Iwabe, Simone; Swider, Malgorzata; Lisi, Brianna; Sumaroka, Alexander; Marinho, Luis Felipe; Appelbaum, Tatyana; Rossmiller, Brian; Hauswirth, William W; Jacobson, Samuel G; Lewin, Alfred S; Aguirre, Gustavo D; Beltran, William A
