通过患者来源的视网膜类器官模型来研究色素性视网膜炎。
Modeling retinitis pigmentosa through patient-derived retinal organoids.
机构信息
Beijing Institute of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing Ophthalmology & Visual Sciences Key Laboratory, Beijing, 100730 China.
School of Ophthalmology and Optometry, The Eye Hospital, Wenzhou Medical University, Wenzhou 325027 China.
出版信息
STAR Protoc. 2021 Apr 8;2(2):100438. doi: 10.1016/j.xpro.2021.100438. eCollection 2021 Jun 18.
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 . 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).
人诱导多能干细胞（hiPSCs）可分化为结构良好的视网膜类器官。在本方案中，我们成功地从患者来源的 hiPSCs 中建立了 3D 视网膜，并构建了色素性视网膜炎模型。此外，通过 CRISPR-Cas9 基因编辑纠正了色素性视网膜炎 GTP 酶调节因子（RPGR）基因突变，挽救了 3D 视网膜的结构和功能。有关本方案使用和执行的完整详细信息，请参阅 Deng 等人（2018 年）。
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