Combining gene editing and anti-inflammatory therapy to treat dry AMD

Dr Jian Liu, University of Bristol - £24,946

Dry age-related macular degeneration (AMD) currently has no effective treatment, but this study is exploring a promising new two-step approach. Researchers are testing whether combining gene editing with an existing anti-inflammatory drug could protect the cells that support vision. By boosting a key protective protein in the retina, this project could open the door to a powerful new treatment strategy for dry AMD.

What is the problem?

The retinal pigment epithelium (RPE) is a vital layer of cells in the retina that supports the light-sensing photoreceptors. Its health is crucial for good vision. Research shows that levels of IRAK-M, an anti-inflammatory protein, are lower in the RPE of older people, and even lower in patients with AMD. Without enough IRAK-M, inflammation goes unchecked, damaging the RPE and, in turn, the retina.

What are they doing?

Previous studies suggest that increasing the production of IRAK-M can help protect against retinal degeneration.

Using mice that lack the IRAK3 gene (and therefore cannot produce IRAK-M), this project will induce chronic inflammation using a high-fat diet. The team will explore three potential interventions:

• Restoring the missing IRAK3 gene using gene therapy
• Reducing inflammation in the RPE using metformin, a safe, inexpensive and widely used anti-inflammatory drug
• Combining both approaches to test for enhanced protection

How can this help?

This research explores whether combining gene therapy with an anti-inflammatory treatment could slow or stop the progression of dry AMD. If successful, it could lay the foundation for a new treatment strategy that targets the root causes of inflammation and degeneration in the retina.