About This Project

Retinal pigment epithelium (RPE) degeneration is a key feature of age-related macular degeneration (AMD), which is the leading cause of vision loss in elder populations (about one-third of people over 80 have AMD). There is no cure for AMD as at to date, and the current anti-AMD drugs only target the neovascularization symptom of this disease. We plan to develop a gene therapy approach to prevent RPE degeneration and AMD. Success of the project will benefit 30 million people globally with AMD.

Ask the Scientists

What is the context of this research?

Recent work from our group at Tulane University and others has discovered novel mechanisms underlying RPE degeneration. With aging, RPE cells are subjected to increasing oxidative stress and other stresses, which lead to RPE degeneration. Mitochondria and lysosomes in the RPE cells are important in maintaining RPE function under stress conditions. Specifically, our preliminary data suggest that a mitochondrial protein PGAM5 plays a pivotal role in RPE degeneration by regulating mitochondrial function. Here we propose that using gene therapy targeting PGAM5 would rejuvenate mitochondrial function under stress conditions, and therefore prevent RPE degeneration.

What is the significance of this project?

In the USA alone, about 1.8 million individuals are afflicted with AMD disease, and that number is projected to reach ∼3 million by 2020, due to the increasing aging population. Therefore, the social-economic impact of AMD and other blinding eye diseases is huge. Based on a survey from American Foundation for the Blind (AFB), Americans fear impact of vision loss more than cancer, HIV/AIDS, heart disease, and stroke. Unfortunately, there is no cure for AMD currently. Our proposal is to address the impressing needs for the society.

The novel point of this project is to target one gene that is important for several fundamental pathophysiological processes in aging and AMD. Other diseases sharing similar mechanisms could also potentially benefit from this approach.

What are the goals of the project?

We will use the funds to purchase the antibodies and other reagents from biomedical companies, transgenic mice from Jackson Lab, as well as paying for confocal microscopy fee and other instruments fee.

Through this study, people will get more understanding about the mechanisms and therapeutics of RPE degeneration. We also plan to share our research finding by publishing our research findings in peer-reviewed journals.