Development of a translatable gene augmentation therapy for CNGB1-retinitis pigmentosa
开发一种可转化的基因增强疗法用于治疗 CNGB1 基因突变引起的视网膜色素变性
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2023.04.005
Occelli, Laurence M; Zobel, Lena; Stoddard, Jonathan; Wagner, Johanna; Pasmanter, Nathaniel; Querubin, Janice; Renner, Lauren M; Reynaga, Rene; Winkler, Paige A; Sun, Kelian; Marinho, Luis Felipe L P; O'Riordan, Catherine R; Frederick, Amy; Lauer, Andreas; Tsang, Stephen H; Hauswirth, William W; McGill, Trevor J; Neuringer, Martha; Michalakis, Stylianos; Petersen-Jones, Simon M