Article
Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients.
Stem cell reports - 10 Apr 2018
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, Jin Zi-Bing
Abstract excerpt
Retinitis pigmentosa (RP) is an irreversible, inherited retinopathy in which early-onset nyctalopia is observed. Despite the genetic heterogeneity of RP, RPGR mutations are the most common causes of this disease. Here, we generated induced pluripotent stem cells (iPSCs) from three RP patients with different frameshift mutations in the RPGR gene, which were then differentiated into retinal pigment epithelium (RPE)...
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