Mutation Independent Gene Therapy (MInT) for Photoreceptor Rescue in Retinal Dystrophies

Retinal dystrophy manifests due to genetic alterations and results in childhood blindness. Replacement of the altered gene with a normal copy (gene therapy), has been efficient only for a short-term in humans. This is because, while each retinal disease has its own genetic mutation, the common disease pathology, i.e, the continuous loss of cells that are responsible for visual perception (photoreceptors), is not addressed at all, by the current approaches.  This project proposes a strategy that will enhance photoreceptor survival; and this will be tested in a mice model of blindness. If successful, this project will help identify a novel approach to treat a wide variety of retinal diseases.