Reduction of retinal ganglion cell death in mouse models of familial dysautonomia using AAV-mediated gene therapy and splicing modulators
使用 AAV 介导的基因治疗和剪接调节剂减少家族性自主神经功能紊乱小鼠模型中的视网膜神经节细胞死亡
期刊:Scientific Reports
影响因子:3.8
doi:10.1038/s41598-023-45376-w
Anastasia Schultz, Shun-Yun Cheng, Emily Kirchner, Stephanann Costello, Heini Miettinen, Marta Chaverra, Colin King, Lynn George, Xin Zhao, Jana Narasimhan, Marla Weetall, Susan Slaugenhaupt, Elisabetta Morini, Claudio Punzo, Frances Lefcort
