Grant: Project Grant

As their name implies, the photoreceptors at the back of the eye capture light signals and turn those into electrical stimuli that are then transmitted to the brain by a network of neurons, enabling vision. When the photoreceptors die, vision is lost and this ultimately results in complete blindness. In the developed world, the most prevalent causes of blindness are due to loss of photoreceptors and include age-related macular degeneration, retinitis pigmentosa, and diabetic retinopathy. With no cures available to directly save the photoreceptors from degeneration, there is a real need for a new approach. We propose to save the photoreceptors from dying, thereby overcoming blindness. Specifically, we propose to protect the photoreceptors from dying by delivering pro-survival factors. As the photoreceptors are located at the back of the eye, in the retina, accessing them is non-trivial. Thus, we will design a local delivery vehicle that will enable sustained release of these pro-survival factors. To realize our goals of repairing the retina to restore vision, we will build on over a decade of research success and the collective experience of our team. We will advance materials that we recently invented specifically for the eye and will now use those materials to deliver protein therapeutics to the photoreceptors. We will investigate novel ways to deliver proteins directly to the retina using a new strategy that we invented based on affinity release. We will test our strategies first in vitro and then in vivo in models of blindness. In advancing knowledge, we will train the next generation of scientists and work together to identify the optimal combination strategy to restore vision in models of blindness, with the ultimate goal of advancing knowledge towards clinical application.