Investigating protein interactions in Doyne’s honeycomb retinal dystrophy and AMD

Dr Michael O’Hare - Queen’s University, Belfast - £25,000

The research project explores how a protein called EFEMP1 may contribute to the growth of leaky blood vessels in wet age-related macular degeneration (AMD) and Doyne’s honeycomb retinal dystrophy. By testing whether existing approved drugs can block this harmful interaction, the study aims to identify a potential new treatment that could be repurposed to help people affected by these conditions.

What is the problem?

In wet AMD and Doyne’s honeycomb retinal dystrophy, a protein called EFEMP1 is produced at unusually high levels and builds up in the retina. Researchers believe this excess EFEMP1 may encourage the growth of abnormal, leaky blood vessels by binding to another protein called TIMP3.

TIMP3 normally helps to stabilise blood vessels and prevent unwanted growth. When EFEMP1 binds to TIMP3, it may stop TIMP3 from doing its job properly, potentially contributing to the development of sight damage.

What are they doing?

This project will test several small-molecule drugs that are already approved to treat other conditions. The researchers will assess whether these drugs can prevent EFEMP1 from binding to TIMP3.

They will also measure whether blocking this interaction reduces the growth of abnormal blood vessels, which is a key feature of wet AMD.

How can this help?

If successful, this research could identify an existing drug that can be repurposed as a treatment for wet AMD and Doyne’s honeycomb retinal dystrophy. Repurposing approved drugs can significantly shorten the time needed to develop new treatments, potentially bringing effective therapies to patients more quickly.