Modeling retinitis pigmentosa through patient-derived retinal organoids

Summary 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).
Graphical abstract
Highlights • Patient-specific human induced pluripotent stem cells (hiPSCs) were established. • The RPGR mutation was corrected by CRISPR/Cas9 in hiPSCs. • hiPSCs were differentiated into 3D retinal organoids.
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, CRISPR-Cas9-mediated correction of the retinitis pigmentosa GTPase regulator mutation rescued the structure and function of the 3D retinae.