Grant: Project Grant

We study a particularly prevalent disease, known as age-related macular degeneration. The macula is the centre portion of the retina, a thin layer of cells and tissue at the back of the eye. The cells which degenerate and die in the macula are called 'cones', a type of photoreceptors responsible for high acuity colour vision. Without these cones, individuals lose their central vision, and they can no longer read, drive or navigate in the environment. Mechanistically, photoreceptors of the eye respond to light, convert this light information into electrical signals and transmit these signals through the retinal layers to the optic nerve, a bundle of about a million of nerve fibres, which connects different parts of the brain, ending in the visual cortex. The brain converts the information collected by the photoreceptors of the eye to create vision. The loss of photoreceptors in the retina is a devastating event because without them we cannot see, and these cells cannot be replaced naturally. Millions of people worldwide have eye diseases in which their photoreceptors die, resulting in vision loss and blindness. For the next five years, our goal is to generate new gene and cell-based therapies to replace these lost photoreceptors. We will also address the challenge of therapeutic access to the retina. The retina is protected by a blood-retinal-barrier, which impedes the delivery of therapeutics when they are administered in the bloodstream. We will test the efficacy of using ultrasound waves to transiently permeate this barrier so that therapies injected into the blood can pass through the barrier and enter the retina. Such an approach has already been used in the brain, and we have preliminary evidence that it will work in the retina. The data obtained through this research will inform on the best strategy to pursue for translation to human patients affected by age-related macular degeneration.