Targeting vascular endothelial growth factor using retinal gene therapy

Area of Science:

• Ophthalmology
• Molecular Biology
• Biotechnology

Background:

• Vascular endothelial growth factor (VEGF) therapies have transformed neovascular retinal disease management.
• Current treatments face challenges including frequent injections, high costs, and treatment resistance.

Purpose of the Study:

• To review current anti-VEGF therapies and emerging treatments.
• To examine advancements in anti-VEGF gene therapy.
• To explore CRISPR-Cas9 for ocular angiogenesis.

Main Methods:

• Review of current pharmacotherapies targeting VEGF.
• Analysis of novel biological therapies and delivery systems.
• Examination of gene therapy advancements and genome editing strategies.

Main Results:

• Newer anti-angiogenic therapies aim for sustained, longer-lasting treatments.
• Gene therapy shows promise for pathologic angiogenesis, with improved delivery and safety.
• CRISPR-Cas9 offers a precise, potentially curative approach by targeting pro-angiogenic factors at the DNA level.

Conclusions:

• Emerging therapies address limitations of current anti-VEGF treatments.
• Gene therapy and genome editing represent significant progress in treating neovascular retinal disorders.
• CRISPR-Cas9 holds potential for permanent suppression of ocular angiogenesis.