Retinal degenerative diseases are disabling conditions affecting the vision of a significant number of Canadians. Acquired conditions, such as age-related macular degeneration (AMD) or diabetic retinopathy, are the leading causes of irreversible blindness in senior and working-age adults, respectively. The prevalence of these conditions is on the rise. Treatments are aimed at slowing the progression of vision loss, but do not represent a regenerative approach to retinal repair. While less prevalent, inherited retinal disorders (IRDs) affect an estimated 90,000 Canadians, with a total cost of disease – including healthcare costs, productivity and well-being – at upwards of $6.7 billion. IRDs include conditions such as retinitis pigmentosa, Stargardt disease, Leber congenital amaurosis, choroideremia, cone (and cone-rod) dystrophy, and achromatopsia. With rare exception, treatments do not exists for inherited conditions.
New therapies for retinal degeneration are focused on the next generation of regenerative medicines. These include gene and cell-based therapeutics, including stem cells. Several of these approaching are already being applied in clinical trials and therapies. While gene therapy has the potential to correct the underlying mechanism of disease in monogeneic disorders, it depends on the presence of viable light-sensitive cells. Stem cell therapy has the potential to replace the light-sensitive photoreceptors lost in later-stage disease, when patients have suffered significant vision loss. Cell-based therapies hold promise for both IRDs and acquired conditions, and discovery in this area is the central aim of the Ballios Lab.