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Modeling retinitis pigmentosa through patient-derived retinal organoids.

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机构: [1]Beijing Institute of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing Ophthalmology & Visual Sciences Key Laboratory, Beijing, 100730 China. [2]School of Ophthalmology and Optometry, The Eye Hospital, Wenzhou Medical University, Wenzhou 325027 China. [3]The First Affiliated Hospital of Chongqing Medical University, Chongqing Key Laboratory of Ophthalmology, and Chongqing Eye Institute, Chongqing 400016, China.
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Human-induced pluripotent stem cells (hiPSCs) can be differentiated into well-structured retinal organoids. In this protocol, we successfully established 3D retinae from patient-derived hiPSCs and built the retinitis pigmentosa model in vitro. Moreover, mutation in the retinitis pigmentosa GTPase regulator (RPGR) gene was corrected by CRISPR-Cas9 gene editing, which rescued the structure and function of the 3D retinae. For complete details on the use and execution of this protocol, please refer to Deng et al. (2018).© 2021 The Author(s).

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Q4 BIOCHEMICAL RESEARCH METHODS

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第一作者机构: [1]Beijing Institute of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing Ophthalmology & Visual Sciences Key Laboratory, Beijing, 100730 China. [2]School of Ophthalmology and Optometry, The Eye Hospital, Wenzhou Medical University, Wenzhou 325027 China.
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