I am a translational scientist with an interest in developing novel therapeutics for retinal diseases. My research has focused on characterizing animal models of inherited retinal disease and treating these animal models with gene therapies, cell therapies or neuroprotective agents. I have had extensive training in molecular cell biology, vision science and clinical and translational science, which has provided me with the expertise, motivation and experience necessary to carry out the proposed research project.

While pursuing a Ph.D. in Biomedical Science under the guidance of Dr. William Hauswirth, I developed a passion for generating treatments for retinal degeneration. My dissertation work included using dual AAV vector technology to deliver a large cDNA, CEP290, to the photoreceptors of the rd16/Nrl-/- mouse, a model of Leber’s Congenital Amaurosis. In addition to my bench work, I sought out opportunities to learn more about the inherited retinal degeneration patient population.

I applied to the Clinical and Translational Science Institute at the University of Florida to receive training in translational science. I received guidance from Dr. Christine Kay as we investigated the relationship between retinal dystrophy patients’ subjective quality of cone- and rod-mediated vision and their electrophysiological performance. To further enhance my understanding of retinal dystrophy patients, Dr. Kay and I traveled to the Bascom Palmer Eye Institute to shadow Dr. Byron Lam, the Chicago Lighthouse to shadow Dr. Gerald Fishman and the Casey Eye Institute to shadow Dr. Mark Pennesi and Dr. Richard Weleber.

Due to my rich engagement with inherited retinal dystrophy patients and expertise in evaluating therapies in animal models, I initiated my post-doctoral training with Dr. Mark Pennesi at the Casey Eye Institute. Under the mentorship of Dr. Pennesi, we evaluated neuroprotective agents in a light-induced retinopathy model and explored neuroprotective mechanisms that can be translated to inherited retinal disease. In addition, we started a collaboration with Dr. Gaurav Sahay to develop lipid nanoparticle gene therapies for retinal degenerations. Dr. Sahay and I initiated our collaboration by characterizing the retinal cell transfection of the most clinically advanced lipid nanoparticle. We quickly realized that our lipid nanoparticles efficiently transfected retinal pigment epithelium, but failed to transfect photoreceptors, which is essential for photoreceptor degenerations. After years of generating novel lipid nanoparticles, we have finally had success at targeting photoreceptors and are optimistic their transfection will translate to primate models.

As I transitioned to faculty, I have been collaborating with Martha Neuringer, Ph.D., at the Oregon National Primate Research Center to develop nonhuman primate models of retinal degeneration and evaluate the retinal transduction efficiency of both AAV and LNP gene therapies. I have managed multiple, collaborative, gene and cell therapy studies at the primate center including 1) evaluating barcoded AAV capsids after intravitreal delivery, 2) evaluating the survival of human central nervous system stem cells after subretinal transplantation, 3) evaluating the efficacy of gene delivery to the retina using ultrasound, and 4) determining if hypoimmune RPE can evade the immune response after subretinal cell transplantation. Most recently, I was awarded the ONPRC Pilot grant and was one of the first groups to subretinally deliver lipid nanoparticles in non-human primates.