Investigation of Novel Pathogenesis and Therapeutic Strategy for AMD

Age-related macular degeneration is the leading cause of irreversible vision loss among elderly people in developed countries. The “dry” form of AMD accounts for 85% of all cases without effective treatment, while the “wet” form occurs in about 15% of the advanced AMD cases, which is being treated with anti-VEGF but not effective in all cases. Our study will identify the factor(s) contributing to the progression of AMD and explore method to halt or reverse AMD retinal lesion. Overall goal is to gain a better understanding of the molecular mechanism of this disease and to develop novel effective therapies.

The goal of our project is to explore novel gene therapy strategies and investigate the molecular mechanisms for the progression of age-related macular degeneration (AMD). During the progression of AMD, a small retina lesion gradually expands to a large lesion.  We have found that the sick retinal pigment epithelial (RPE) cells can secrete factors that make the neighboring healthy RPE cells dysfunctional and promote the progression of AMD. Our first aim will be to isolate and identify these factors. Our second aim will be to use an adeno-associated virus (AAV) vector to deliver a gene intended to rescue the retina or block the AMD-like pathology. To date, our research has been unique because it will explore the molecular mechanism for AMD progression and novel gene therapy strategies to halt the progression of AMD. A successful gene therapy for AMD has not been trialed before, therefore our research has exciting potential for patients suffering from this debilitating condition. By identifying new factors responsible for the progression of AMD, we will also inspire future research.