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Updated: Jul 8, 2025
06:48
Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
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Genes as drugs for glaucoma: latest advances
Teresa Borrás1, Michelle Stepankoff2, John Danias2
1University of North Carolina at Chapel Hill, North Carolina.
Current Opinion in Ophthalmology
|December 20, 2023
Summary
Gene therapy shows promise for glaucoma treatment, with preclinical studies identifying genes that reduce intraocular pressure and protect retinal cells. Several companies are advancing these gene therapies toward clinical development.
Area of Science:
- Ophthalmology
- Genetics
- Molecular Biology
Background:
- Glaucoma is a leading cause of irreversible blindness worldwide, characterized by elevated intraocular pressure (IOP) and retinal ganglion cell (RGC) neurodegeneration.
- Current treatments primarily focus on lowering IOP but do not fully prevent disease progression or vision loss.
Approach:
- This review summarizes recent advances in gene therapy for glaucoma, focusing on preclinical findings and potential therapeutic targets.
- Identified genes (e.g., MMP3, PLAT, IκB, GLIS, SIRT, Tie-2, AQP1, BDNF, C3, TGFβ, MYOC, ANGPTL7) demonstrate efficacy in reducing IOP and providing neuroprotection in experimental models.
Key Points:
- Multiple genes have shown potential in preclinical glaucoma models to lower intraocular pressure and protect retinal ganglion cells.
- These genes target diverse mechanisms involved in glaucoma pathogenesis.
- Several promising gene candidates are progressing towards drug development pipelines.
Conclusions:
- Significant preclinical research is underway, with numerous biotechnology and pharmaceutical companies actively developing gene therapies for glaucoma.
- The substantial global market for glaucoma treatments suggests a growing interest and investment in novel gene-based therapeutic strategies.
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