Our group is focused on restoring sight to the blind. There are many types of retinal disease, including glaucoma and macular degeneration, that lead to loss of sight from the death of the cells in the light-sensitive part of the eye caled the retina. We have found that non-mammalian vertebrates, like frogs and fish, can regenerate new cells to replace those lost from disease, but mammals, like ourselves, cannot. We have discovered key genes that are needed for regeneration in lower vertebrates, and we have found that putting these genes into mice can restore some regeneration potential. Our ongoing studies are aimed at stimulating more effective regeneration in the retina to restore vision in people that have lost their sight.
We use cell culture models and live mice to examine the genes that are needed for regeneration in the retina. Our methods of analysis include immunofluorescence analysis of retina, electron microscopic studies (with serial block face scanning EM in particular), FACS purifying retinal cell populations and single cell RNAseq, epigenetic analyses (ATAC-seq and ChIP-seq) and CRISPR screening. Our work is now primarily in mice, but in the past we have also used fish, frogs and birds, taking a comparative view of regeneration.
It is our hope that we will one day restore sight in patients. At present, we are developing a viral gene therapy that will stimulate this process in patients with vision loss from trauma or retinal stroke.