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Updated: Jun 17, 2026

Directed Induction of Retinal Organoids from Human Pluripotent Stem Cells
Published on: April 21, 2021
Retinal Organoids: Innovative Tools for Understanding Retinal Degeneration
Nadia Galindo-Cabello1,2, Estefanía Caballano-Infantes3, Gregorio Benites2
1Department of Cell Biology, Genetics, Histology and Pharmacology, Faculty of Medicine, University of Valladolid, 47003 Valladolid, Spain.
Abstract:
Retinal degenerative diseases (RDDs) comprise diverse genetic and phenotypic conditions that cause progressive retinal dysfunction and cell loss, leading to vision impairment or blindness. Most RDDs lack appropriate animal models for their study, which affects understanding their disease mechanisms and delays the progress of new treatment development. Recent advances in stem cell engineering, omics, and organoid technology are facilitating research into diseases for which there are no previously existing models. The development of retinal organoids produced from human stem cells has impacted the study of retinal development as well as the development of in vitro models of diseases, opening possibilities for applications in regenerative medicine, drug discovery, and precision medicine. In this review, we recapitulate research in the retinal organoid models for RDD, mentioning some of the main pathways underlying retinal neurodegeneration that can be studied in these new models, as well as their limitations and future challenges in this rapidly advancing field.
Insights
Human stem cell-derived retinal organoids offer new models for studying retinal degenerative diseases (RDDs). These advanced models aid in understanding disease mechanisms and developing treatments for vision loss.
Area of Science:
- Biomedical research
- Regenerative medicine
- Ophthalmology
Background:
- Retinal degenerative diseases (RDDs) cause progressive vision loss and blindness.
- Lack of adequate animal models hinders understanding RDD mechanisms and treatment development.
Purpose of the Study:
- To review the use of retinal organoids derived from human stem cells for studying RDDs.
- To discuss the potential of these models in understanding neurodegeneration and advancing precision medicine.
Main Methods:
- Utilizing stem cell engineering and organoid technology to create in vitro models of RDDs.
- Recapitulating research on retinal organoid models for various RDDs.
Main Results:
- Retinal organoids provide valuable in vitro models for studying retinal development and disease.
- These models facilitate the investigation of neurodegenerative pathways in RDDs.
Conclusions:
- Retinal organoids represent a significant advancement for RDD research, offering new avenues for drug discovery and regenerative medicine.
- Future challenges include refining models and addressing limitations for broader clinical application.

