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Updated: Oct 15, 2025
Generation of Retinal Organoids from Healthy and Retinal Disease-Specific Human-Induced Pluripotent Stem Cells
Published on: December 9, 2022
Retinal organoids as models for development and diseases
Xiao Zhang1, Wen Wang1, Zi-Bing Jin2
1Beijing Institute of Ophthalmology, Beijing Tongren Eye Center, Beijing Tongren Hospital, Capital Medical University, Beijing Ophthalmology & Visual Science Key Laboratory, Beijing, 100730, China.
Abstract:
The evolution of pluripotent stem cell-derived retinal organoids (ROs) has brought remarkable opportunities for developmental studies while also presenting new therapeutic avenues for retinal diseases. With a clear understanding of how well these models mimic native retinas, such preclinical models may be crucial tools that are widely used for the more efficient translation of studies into novel treatment strategies for retinal diseases. Genetic modifications or patient-derived ROs can allow these models to simulate the physical microenvironments of the actual disease process. However, we are currently at the beginning of the three-dimensional (3D) RO era, and a general quantitative technology for analyzing ROs derived from numerous differentiation protocols is still missing. Continued efforts to improve the efficiency and stability of differentiation, as well as understanding the disparity between the artificial retina and the native retina and advancing the current treatment strategies, will be essential in ensuring that these scientific advances can benefit patients with retinal disease. Herein, we briefly discuss RO differentiation protocols, the current applications of RO as a disease model and the treatments for retinal diseases by using RO modeling, to have a clear view of the role of current ROs in retinal development and diseases.
Insights
Retinal organoids (ROs) derived from pluripotent stem cells offer promising models for studying retinal development and diseases. Further development is needed to enhance their accuracy and utility for novel therapeutic strategies.
Area of Science:
- Stem cell biology
- Ophthalmology
- Regenerative medicine
Background:
- Pluripotent stem cell-derived retinal organoids (ROs) are emerging models for retinal research.
- These three-dimensional (3D) models offer potential for studying retinal development and disease mechanisms.
- Current limitations include a lack of standardized quantitative analysis across differentiation protocols.
Purpose of the Study:
- To review the current state of retinal organoid technology.
- To discuss RO differentiation protocols and their applications in disease modeling.
- To highlight the potential of ROs for developing novel therapeutic strategies for retinal diseases.
Main Methods:
- Review of existing literature on retinal organoid differentiation and applications.
- Discussion of genetic modification and patient-derived ROs for disease simulation.
- Analysis of current quantitative technologies for RO assessment.
Main Results:
- Retinal organoids show significant promise as preclinical models for retinal diseases.
- Patient-derived and genetically modified ROs can mimic disease-specific microenvironments.
- A universal quantitative analysis technology for ROs is still lacking.
Conclusions:
- Retinal organoids are valuable tools for understanding retinal development and disease.
- Improving differentiation efficiency, stability, and inter-model consistency is crucial.
- Further advancements are essential for translating RO research into effective patient treatments.
