Using patient-derived iPSCs to model and treat inherited retinal degenerative blindness

Inherited retinal degenerative disorders such as retinitis pigmentosa are characterized by death of the light sensing photoreceptive neurons of the outer retina. Like the rest of the CNS, the retina has little capacity for endogenous regeneration, and as a result, photoreceptor cell death causes debilitating irreversible blindness. Gene augmentation has the potential to prevent photoreceptor cell death, while cell replacement could actually repopulate the retina with new functioning photoreceptor cells and restore vision. In this talk I will show how we are using patient-specific iPSCs to evaluate disease pathophysiology, test novel gene-based therapeutics and develop autologous photoreceptor cell replacement for the treatment of retinal degenerative blindness.