Modeling retinitis pigmentosa through patient-derived retinal organoids

Yan-Ping Li , Wen-Li Deng , Zi-Bing Jin 
Source: STAR Protoc
Publication Date: (2021)
Issue: 2(2): 100438
Research Area:
Dermatology/Tissue Engineering
Stem Cells
Gene Expression
Regenerative medicine
Cells used in publication:
SMC, bladder (BdSMC), human
Species: human
Tissue Origin: bladder
Induced Pluripotent Stem Cell (iPS), human
Species: human
Tissue Origin:
Endothelial, MV bladder, human
Species: human
Tissue Origin: bladder
Nucleofector® I/II/2b
4D-Nucleofector® X-Unit

Please see protocol: reprogramming of urinary cells ( T-020? ); genome editing of iPSCs by transfecting px330 plasmid with P3 solution and CA-137 


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).