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Updated: May 6, 2026
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Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
Published on: March 16, 2022
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The gene therapy revolution in ophthalmology
1College of Pharmacy, Salman Bin Abdulaziz University, Al-Kharj, Saudi Arabia.
Summary
Gene therapy shows promise for treating inherited eye diseases like retinitis pigmentosa and LCA using adeno-associated virus (AAV) vectors in animal models. Clinical trials are advancing for these and other conditions.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Gene therapy offers a promising avenue for treating various ophthalmic conditions.
- Significant research has been conducted using animal models to explore its efficacy.
Purpose of the Study:
- To review the application of gene therapy, particularly using adeno-associated virus (AAV) vectors, in animal models for inherited retinal diseases.
- To highlight progress in treating conditions such as retinitis pigmentosa, Leber's Congenital Amaurosis (LCA), and Stargardt disease.
Main Methods:
- Utilizing adeno-associated virus (AAV) vectors to deliver functional genes in mouse and canine models.
- Employing gene correction strategies for specific mutations causing retinitis pigmentosa, LCA, and Stargardt disease.
- Investigating gene therapy for other conditions like red-green color blindness and primary open-angle glaucoma (POAG) in relevant animal models.
Main Results:
- Successful correction of gene mutations and functional improvements in animal models for retinitis pigmentosa, LCA (LCA2), and Stargardt disease.
- Demonstrated efficacy of AAV vectors in restoring visual function and retinal health.
- Positive outcomes in treating red-green color blindness in non-human primates and potential for AMD and POAG.
Conclusions:
- Gene therapy, especially with AAV vectors, is a viable and effective strategy for treating a range of ophthalmic diseases.
- Animal model studies provide strong evidence for the potential of gene therapy in human clinical applications.
- Advancements are paving the way for upcoming clinical trials and broader therapeutic applications in ophthalmology.
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