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Toxicity Screens in Human Retinal Organoids for Pharmaceutical Discovery

Toxicity Screens in Human Retinal Organoids for Pharmaceutical Discovery

Published on: March 4, 2021

Inherited Retinal Diseases and Retinal Organoids as Preclinical Cell Models for Inherited Retinal Disease Research.

Kristen E Ashworth^1,2, Jessica Weisbrod², Brian G Ballios^1,2,3

¹Institute of Medical Science, University of Toronto, Toronto, ON M5S 3H2, Canada.

|June 27, 2024

Summary

Inherited retinal diseases (IRDs) cause irreversible vision loss. Patient-derived retinal organoids offer a promising human model for understanding IRD pathogenesis and accelerating new treatment discoveries.

Keywords:

gene therapy inherited retinal diseases neurodegeneration retinal organoid retinitis pigmentosa stem cell therapy

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Generation of Retinal Organoids from Healthy and Retinal Disease-Specific Human-Induced Pluripotent Stem Cells

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Published on: December 9, 2022

Area of Science:

Ophthalmology
Genetics
Regenerative Medicine

Background:

Inherited retinal diseases (IRDs) are a diverse group of genetic conditions causing progressive photoreceptor degeneration and irreversible vision loss.
Current treatments for IRDs are limited, highlighting the need for advanced research models to accelerate therapeutic development.

Purpose of the Study:

To review the development and significance of retinal organoids as human-based in vitro models for studying the retina.
To explore the application of retinal organoids in understanding IRD pathologies and advancing treatment discovery.

Main Methods:

Review of existing literature on retinal organoid development and applications in IRD research.
Discussion of retinal organoids as patient-derived models for investigating disease mechanisms and therapeutic strategies.

Main Results:

Retinal organoids provide a valuable human in vitro model for studying retinogenesis and retinal pathologies.
Patient-derived retinal organoids enable precise investigation of IRD pathogenesis and variant-specific mechanisms.

Conclusions:

Retinal organoids are crucial for advancing the understanding of inherited retinal diseases.
These organoids hold significant promise for the future of IRD treatment discovery and development.