Center for Orphan Disease Research & Therapy

Jean Bennett, M.D., Ph.D

Jean Bennett, MD PhDKirby Professor of Ophthalmology, Cell and Developmental Biology

Dr. Bennett research interests focuses on Molecular Genetics of retinal degenerations and Gene therapy-mediated treatment of ocular disease. Dr. Bennett studies the molecular genetics of inherited retinal degenerations with the idea of using this knowledge to develop rational approaches for treatment of diseases, such as retinitis pigmentosa and age-related macular degeneration.

Studies in Dr. Bennett laboratory range for identifying the molecular bases of retinal degenerations, generating animal models for these diseases and rescuing vision in animal models through gene based treatments. Dr. Bennett was one of the first investigators to use viral vectors to deliver transgenes to specific cells in the retina and also led the first team to demonstrate proof-of-principle of ocular gene therapy. She has developed a number of strategies for gene therapy-mediated treatments for retinal disease. Dr. Bennettís work leads naturally to translational research. For example, a recent study conducted in her lab and with collaborators at UPenn, Cornell and University of Florida led to a remarkable reversal of blindness in a canine model of a blinding disease affecting infants. This treatment will be tested in a human clinical trial.

We are interested in the identification and characterization of genes that are defective in blinding and currently untreatable hereditary retinal degenerations such as retinitis pigmentosa, macular degeneration and choroideremia. In order to gain an understanding of the pathogenetic mechanisms which lead to these diseases, we generate and study animal models for the diseases. We are also interested in intervening with the progression of retinal degeneration by introducing corrective genes or molecules which delay programmed cell death into the retina. For this purpose, the laboratory has developed virus-based methods for delivering functional genes into differentiated retinal photoreceptors, retinal pigment epithelial, trabecular meshwork, iris and corneal endothelial cells. The viruses are being used to test the possibility of genetic therapy in spontaneous and genetically engineered animal models with retinal degeneration. Additional studies focus on the immune system's response to adenovirus-mediated gene delivery to the retina.

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