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Updated: Jun 28, 2026

Toxicity Screens in Human Retinal Organoids for Pharmaceutical Discovery
Published on: March 4, 2021
Testing retinal toxicity of drugs in animal models using electrophysiological and morphological techniques
1Department of Physiology and Biophysics, Ruth and Bruce Rappaport Faculty of Medicine, Technion-Israel Institute of Technology and Rappaport Institute, P.O. Box 9649, Haifa, 31096, Israel. iperlman@tx.technion.ac.il.
Abstract:
Drugs are frequently tested for retinal toxicity in animal models in order to address applied and basic research questions. When a retinal toxicity study is designed, the researcher needs to consider several factors depending on his/her research questions. Among the factors that need to be addressed before a toxicity study is conducted are: the animal species to be used, choice of experimental (functional and/or morphological) techniques, procedure of testing, period of follow-up, and modes of data analysis. This review is a summary of 20 years' experience of studying retinal toxicity of different drugs in rabbits and rats. The use of the electroretinogram and the visual evoked potential for assessment of outer and inner retinal function, respectively, is described as well as the use of morphological techniques (histology, histochemistry, and immunocytochemistry). The advantages and limitations of functional and morphological techniques are discussed with specific examples from my experience. Recommendations for future drug toxicity studies are summarized.
Insights
This review details 20 years of experience in animal models for drug-induced retinal toxicity studies. It covers species selection, functional and morphological testing methods, and data analysis for robust safety assessments.
Area of Science:
- Ophthalmology
- Toxicology
- Pharmacology
Background:
- Drug-induced retinal toxicity is a significant concern in pharmaceutical development.
- Animal models are crucial for evaluating drug safety and understanding ocular side effects.
- Standardized methodologies are needed for reliable assessment of retinal toxicity.
Purpose of the Study:
- To summarize 20 years of experience in studying drug retinal toxicity in animal models.
- To provide guidance on selecting appropriate methodologies for retinal toxicity studies.
- To discuss the advantages and limitations of various functional and morphological assessment techniques.
Main Methods:
- Review of 20 years of research on drug retinal toxicity in rabbits and rats.
- Utilized electroretinogram (ERG) for outer retinal function and visual evoked potential (VEP) for inner retinal function.
- Employed morphological techniques including histology, histochemistry, and immunocytochemistry.
Main Results:
- Detailed description of ERG and VEP applications in assessing drug effects on retinal function.
- Illustrated the utility of histological, histochemical, and immunocytochemical methods for morphological evaluation.
- Discussed specific advantages and limitations of functional versus morphological techniques based on empirical data.
Conclusions:
- Effective retinal toxicity studies require careful consideration of animal species, experimental techniques, and follow-up periods.
- A combination of functional and morphological assessments provides a comprehensive evaluation of drug-induced retinal toxicity.
- Recommendations are provided for optimizing future drug toxicity studies in ophthalmology.

