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Updated: Jun 10, 2025
Generation of Retinal Organoids from Healthy and Retinal Disease-Specific Human-Induced Pluripotent Stem Cells
Published on: December 9, 2022
Retinal Organoids from Induced Pluripotent Stem Cells of Patients with Inherited Retinal Diseases: A Systematic
1Department of Medicine, Seoul National University College of Medicine, 103 Daehak-ro, Jongno-gu, Seoul, 03080, Republic of Korea.
Background:
Currently, most inherited retinal diseases lack curative interventions, and available treatment modalities are constrained to symptomatic approaches. Retinal organoid technology has emerged as a method for treating inherited retinal diseases, with growing academic interest in recent years. The purpose of this review was to systematically organize the current protocols for generating retinal organoids using induced pluripotent stem cells from patients with inherited retinal disease and to investigate the application of retinal organoids in inherited retinal disease research.
Methods:
Data were collected from the PubMed, Scopus, and Web of Science databases using a keyword search. The main search term used was "retinal organoid," accompanied by secondary keywords such as "optic cup," "three-dimensional," and "self-organizing." The final search was conducted on October 2, 2024.
Results:
Of the 2,129 studies retrieved, 130 were included in the qualitative synthesis. The protocols for the generation of retinal organoids in inherited retinal disease research use five major approaches, categorized into 3D and a combination of 2D/3D approaches, implemented with modifications. Disease phenotypes have been successfully reproduced via the generation of retinal organoids from the induced pluripotent stem cells of individuals with inherited retinal diseases, facilitating the progression of research into novel therapeutic developments. Cells have been obtained from retinal organoids for cell therapy, and progress toward their potential integration into clinical practice is underway. Considering their potential applications, retinal organoid technology has shown promise across various domains.
Conclusion:
In this systematic review, we organized protocols for generating retinal organoids using induced pluripotent stem cells from patients with inherited retinal diseases. Retinal organoid technology has various applications including disease modeling, screening for novel therapies, and cell replacement therapy. Further advancements would make this technology a clinically significant tool for patients with inherited retinal diseases.
Insights
Retinal organoid technology offers new hope for inherited retinal diseases by modeling patient-specific conditions and advancing cell therapies. This review details protocols for generating these organoids and their therapeutic potential.
Area of Science:
- Biomedical Engineering
- Stem Cell Biology
- Ophthalmology
Background:
- Inherited retinal diseases currently lack curative treatments, relying on symptomatic management.
- Retinal organoid technology, derived from patient-induced pluripotent stem cells, is a rapidly advancing field for studying these conditions.
- This review systematically organizes protocols and applications of retinal organoids in inherited retinal disease research.
Purpose of the Study:
- To systematically review and organize protocols for generating retinal organoids from patient-derived induced pluripotent stem cells.
- To investigate the diverse applications of retinal organoids in the research of inherited retinal diseases.
Main Methods:
- Systematic literature search of PubMed, Scopus, and Web of Science databases.
- Keywords included "retinal organoid," "optic cup," "three-dimensional," and "self-organizing."
- Qualitative synthesis of 130 selected studies from an initial retrieval of 2,129.
Main Results:
- Five major approaches for retinal organoid generation were identified, utilizing 2D/3D and 3D methods.
- Patient-derived retinal organoids successfully replicate disease phenotypes, aiding therapeutic development.
- Retinal organoids are being explored for cell therapy, with progress toward clinical integration.
Conclusions:
- Protocols for generating patient-derived retinal organoids have been systematically organized.
- Retinal organoid technology shows significant promise for disease modeling, therapeutic screening, and cell replacement therapy.
- Further development of retinal organoid technology could lead to significant clinical tools for inherited retinal diseases.
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