6 Macula Vision Research Foundation Spring/Summer 2017 impact Philanthropy: investing in new treatments and a Cure Over the last twenty years, MVRF has funded 185 grants totaling more than $22 million in retina-specific research. Kazuko nishikura, PhD Professor gene expression and regulation Program the wistar institute invoLvement of rnA eDiting AnD rnA interferenCe in AmD PAthogeneSiS There are two forms of AMD, wet and dry, which are classified based on the presence or absence of blood vessels that disruptively invade the retina, respectively. Recent advancements in understanding wet AMD have led to several FDA-approved therapies. in contrast, there are no approved treatments for dry AmD. With funding from MVRF, we are focused on an emerging idea for dry AMD: the reduction in expression of Dicer, a critical enzyme involved in RNA interference (RNAi), and the accumulation of Alu RNA, are major causes of dry AMD. RNAi is a naturally occurring process that leads to the “silencing” of genes. Alu RNA is transcribed from retrotransposons, reminiscent of ancient viruses that invaded and spread over the human genome. Alu RNA accumulation triggers programmed cell death of retinal pigment epithelium (RPE) cells, revealing a previously unexplored abnormality that underlies the molecular pathogenesis of AMD. Yet why this happens is completely unknown. In our lab, we plan to test this new hypothesis. If true, understanding at least one of the mechanisms that underlies retinal cell death that leads to AMD will be exposed, this would cause a global paradigm shift in preventing and treating AMD. Because of MVRF’s considerable research investment of charitable, private dollars along with the pool of public research dollars through the National Institutes of Health (NIH), the underlying causes of Age-related Macular Degeneration (AMD) are becoming understood. Emerging effective treatments and new gene discoveries are happening in labs all over the world as the best scientific researchers in the world hone in on discoveries that will lead to conquering AMD.