In both RP and AMD, loss of vision is a result of photoreceptor cell degeneration and death. Treatments that can prevent photoreceptor degeneration in experimental models of retinal degenerations may have utility in preventing degeneration in both RP and AMD. I am interested in investigating novel treatments to preserve retinal function in patients with RP. My research collaborator, Matthew M. LaVail demonstrated that subretinal and intravitreal injection of various growth factors protect the retina from degenerating in several models of inherited retinal degenerations. More recently, Dr. LaVail's laboratory has used recombinant adeno-associated virus (AAV) vectors for sustained application of growth factors and ribozymes in models of retinal degenerations. Ribozymes are catalytic RNA molecules that can recognize and cleave other RNA molecules of specific sequences. In the case of a dominantly inherited disease, ribozymes could be designed specifically to reduce the abnormal gene product.Dr. LaVail's laboratory has demonstrated that sub-retinal injection of AAV-ribozyme vectors with rod opsin promoters in transgenic models of retinal degeneration can delay photoreceptor loss and elevate ERG amplitudes. My research is designed to further investigate the retinal functional response to ribozyme therapy in different experimental models of dominantly inherited retinal degenerations.

Patients with rod specific defects undergo degeneration not only of rods, but also of cone photoreceptors. Because cones are responsible for fine visual acuity and color perception, their degeneration is particularly disabling for patients. I am
interested in studying the relationship between cone photoreceptor survival and rod-specific gene defects in hereditary retinal degenerations, with the goal of preserving visual function mediated by both rods and cones.