Update on Gene Therapy for Diabetic Retinopathy

Document Type : Review Article

Authors

1 Ophthalmology, faculty of medicine, Benghazi University, Benghazi, Libya

2 Department of Ophthalmology, Faculty of Medicine, University of Benghazi, Benghazi, Libya

10.21608/asmj.2025.343792.1374

Abstract

Background: Gene therapy has emerged as a promising strategy to alleviate the treatment burden of diabetic retinopathy (DR). The eye is an ideal target for gene therapy due to its immune-privileged status and ease of delivery. Gene therapy relies on the expression of a particular transgene to treat or cure a disease with the fewest possible negative effects. Common gene therapy techniques include gene augmentation, gene-specific targeting, and CRISPR/Cas9-mediated genome editing. Genetic material is typically delivered using viral or non-viral methods. Two gene families have been investigated as possible targets for DR gene therapy: those that address retinal protection and those that target retinal vasculopathy. The first set of genes was chosen with the intention of disrupting the intraocular VEGF pathway in order to reduce neovascularization and increase angiogenesis. Despite significant progress in anti-VEGF therapies, challenges such as the need for frequent intravitreal injections, variable patient response, and failure to address early neurodegeneration in diabetic retinopathy remain unresolved. Gene therapy emerges as a promising alternative by enabling sustained therapeutic effects and targeting multiple pathological pathways. However, clinical translation is hindered by challenges including vector delivery efficiency, immune responses, and off-target effects.
This Review Aims to: Summarize recent advances in gene therapy strategies targeting both angiogenesis and neuroprotection in DR, evaluate key challenges limiting clinical application, and to explore the potential of CRISPR/Cas9 systems in overcoming these barriers.

Keywords

Main Subjects

Files

Share

How to cite

Statistics