The macula is a small, but extremely important area located at the centre of the retina, the light-sensing tissue that lines the back of the eye. It is responsible for seeing fine details clearly. When the cells in the macular are damaged or degenerate the ability to make out finer details both up close and at a distance begins to decline. Usually, only the central vision is affected so people have peripheral vision.
Macular disease affects people of all ages. Age-related macular degeneration is the most common condition, generally affecting people over 50 and diabetic macular oedema is the most common cause of sight loss in diabetics. A group of rare inherited conditions called juvenile macular dystrophies can affect much younger people, including children. Two examples are Stargardt’s Disease and Best Disease.
The study will look at a type of gene therapy called antisense therapy (AON) which aims to correct the fault in the gene that causes Stargardt disease.
In this pilot study, the team will focus on the development of therapeutic approaches by targeting a newly discovered regulatory mechanism to rescue phagocytosis in disease.