Testing 3 new approaches to treatment in a mouse version of macular degeneration

Overview

Age-related macular degeneration (AMD) affects more than half a million people and is the main cause of blindness registrations in the UK in the over-65s. Sight loss in AMD is due to death of the light-detecting cells in the macula – a part of the eye that’s vital for central vision and seeing in detail.

Older age, genetics, the immune system and environmental factors such as smoking all play a part in the risk of getting AMD. This makes it a complex condition that can affect one person very differently to another, or even one eye differently to the other in the same person. It also means it’s hard to find general targets for treatment or a useful way to test the effect of potential new treatments.

In the current project, Dr Shyamanga is spending a year with a team in the USA that has developed a mouse model of AMD based on a genetic fault found in people with a rare inherited form of macular degeneration. The condition looks a lot like AMD, but in humans starts in the 30s and leads to being registered blind in people’s 50s.

The team will be trying 3 approaches to treatment that could be appropriate for different stages of the condition, before symptoms begin, once symptoms have begun but there is still some central vision, and at the latest stage when most of the cells have been lost. The approaches are drug-based, to correct unhealthy activity in the immune system, gene therapy to correct the genetic fault itself, and cell replacement therapy to repair a damaged macula. The aim is to find potential treatments that can stop or slow macular degeneration that could then be taken on to human clinical trials.