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Importance of Nonhuman Primates as a Model System for Gene Therapy Development in Ophthalmology
Fabian Wozar1, Immanuel Seitz1, Felix Reichel1
1University Eye Hospital, University Hospital Tübingen Centre of Ophthalmology, Tübingen, Germany.
Abstract:
Gene therapy is a treatment concept that uses, in most cases, viral vectors to deliver a therapeutic transgene to target cells. Although the idea of gene therapy dates back over 50 years ago, due to the complexity of the treatment concept, it took until the last decade for the responsible agencies like FDA and EMA to recommend the first gene therapy products for clinical use. The development of these therapies relies on molecular engineering of specifically designed vectors and models to test the effectiveness and safety of the treatment. Despite an increasing effort to find effective surrogates, animal models are still irreplaceable in gene therapy development. Rodents are important for exploring pathways and disease mechanisms and identifying potential treatment targets. However, only the primate eye resembles the human eye to a degree where most structures are nearly identical. Some research questions can therefore only be answered using a nonhuman primate (NHP) model. In this review, we want to summarize these key features and highlight the importance of the NHP model for gene therapy development in ophthalmology.
Insights
Gene therapy uses viral vectors for treatment, with nonhuman primate (NHP) models being crucial for ophthalmology research due to eye structure similarities to humans.
Area of Science:
- Ophthalmology
- Gene Therapy
- Translational Medicine
Background:
- Gene therapy, a treatment using viral vectors to deliver therapeutic genes, has seen recent clinical approvals after decades of development.
- Developing effective gene therapies requires rigorous testing of vector safety and efficacy.
- While rodents aid in understanding disease mechanisms, certain research necessitates more complex models.
Purpose of the Study:
- To summarize the key features and importance of nonhuman primate (NHP) models in ophthalmology gene therapy development.
- To highlight why NHP models are irreplaceable for specific gene therapy research questions in vision science.
Main Methods:
- Review of existing literature on gene therapy development and animal models.
- Comparative analysis of ocular structures between humans and nonhuman primates.
- Discussion of research questions addressable only by NHP models in ocular gene therapy.
Main Results:
- Nonhuman primate eyes closely resemble human eyes in structure, making them valuable models.
- NHP models allow for the study of gene therapy delivery and efficacy in a relevant anatomical context.
- Certain complex ocular research questions cannot be adequately addressed by rodent models.
Conclusions:
- Nonhuman primate models are essential for advancing gene therapy in ophthalmology.
- The anatomical similarity of the NHP eye to the human eye supports its use in preclinical gene therapy studies.
- Further utilization of NHP models will accelerate the development of novel ocular gene therapies.
