Grant: Emerging Team Grant: From Genes to Proteins, Cells, Tissues and Patients

The socio-economic burden of visual impairment and blindness is tremendous and will increase dramatically as our population ages. This underscores the urgent need for Canada to develop a comprehensive national vision plan aimed at the treatment and prevention of blinding disorders. Diseases affecting the retina such as retinitis pigmentosa, glaucoma, retinal ischemia and age-related macular degeneration cause visual loss in millions of people in North America alone, and yet little is known about the mechanisms or causes of many of these diseases. What is known, however, is that cell death through apoptosis is the final common pathway for many forms of retinal disease. Our research program has provided compelling evidence showing that a therapy that targets the X-linked inhibitor of apoptosis (XIAP) to the retina can prevent cell death and significantly preserve vision in animal models of retinal ischemia, retinal detachment and in genetic forms of retinitis pigmentosa (RP). With the current Emerging Team Grant, we propose a strategy for the development of XIAP therapy for the treatment of retinal disease. We will establish a multidisciplinary team of basic and clinical scientists with expertise in the basic science of visual structure and function, in anti-apoptotic mechanisms and therapies, in vector delivery to the eye, in animal models of retinal disease, and in the establishment and follow-up of clinical trials. By the end of the five-year granting period, we will have conducted all the studies necessary to bring XIAP therapy to the clinic.