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Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients

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 d...

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書誌詳細
出版年:Stem Cell Reports
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Elsevier 2018
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC5998840/
https://ncbi.nlm.nih.gov/pubmed/29526738
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2018.02.003
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