Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients - Deng, Wen-Li; Gao, Mei-Ling; Lei, Xin-Lan; Lv, Ji-Neng; Zhao, Huan; He, Kai-Wen; Xia, Xi-Xi; Li, Ling-Yun; Chen, Yu-Chen; Li, Yan-Ping; Pan, Deng; Xue, Tian; et al. (Stem cell reports, 2018)
Keywords: Cell Differentiation; Ciliopathies - pathology; Ciliopathies - physiopathology; Ciliopathies - therapy; ciliopathy; cilium; disease modeling; electrophysiology; Eye Proteins - genetics; Genetic Therapy; Humans; Induced Pluripotent Stem Cells - metabolism; Induced Pluripotent Stem Cells - pathology; Mutation - genetics; Organoids - pathology; patient-derived iPSCs; photoreceptor; Photoreceptor Cells - metabolism; Photoreceptor Cells - pathology; Potassium Channels - metabolism; Retina - pathology; retinal organoid; retinitis pigmentosa; Retinitis Pigmentosa - pathology; Retinitis Pigmentosa - physiopathology; Retinitis Pigmentosa - therapy; RPE cells; RPGR; cilium; electrophysiology; patient-derived iPSCs; photoreceptor; retinal organoid; disease modeling; retinitis pigmentosa; RPE cells; RPGR; ciliopathy