Macular Degeneration Research - Current Awards Michael E. Boulton, Ph.D.University of Florida Title: Preprogrammed Bone Marrow Cells as a Systemic Therapy for Dry AMD Co-PI(s):
Maria Grant, M.D.
University of Florida Summary: Age-related macular degeneration is the leading cause of irreversible blindness in the elderly. Dry AMD, which represents 85% of AMD, is associated with retinal pigment epithelial (RPE) dysfunction and death for which there is currently no treatment. The development of a minimally invasive cellular therapy that can be given systemically will a) overcome the need for invasive ocular surgery and b) offer the potential for prevention rather than intervention since this therapy can be given much earlier in the disease. Successful development of this approach will offer an important treatment for the 1.7 million plus Americans who are threatened with visual loss from dry AMD, improve quality of life and reduce social and healthcare costs. We predict that the BrightFocus funding for this proposed preclinical study will provide the necessary data to confirm the efficacy of this cellular therapy and allow this pioneering research to translate into phase 1 and 2 clinical trials. Program: Macular Degeneration  Milam A. Brantley, Jr., M.D., Ph.D.Vanderbilt University Medical Center Title: Quantitative Evaluation of Environmental Risk for AMD Summary: The research is examining how the environment and genetics contribute to age-related macular degeneration (AMD). Using a cutting-edge technique called metabolomics, Dr. Milam Brantley, Jr., and colleagues are measuring blood levels of thousands of markers of metabolism (metabolites) to identify the environmental influences on the risk of developing AMD. Determining the combination of metabolism and genetics associated with AMD will lead to a better understanding of the disease and, ultimately, may lead to personalized clinical care. Program: Macular Degeneration  Jiyang Cai, Ph.D.University of Texas Medical Branch Title: Exosomal microRNA from the RPE Summary: Interactions between the retinal pigment epithelium (RPE,) the cell layer that nourishes and detoxifies the retina, and their underlying choroidal blood vessels are critical in maintaining the structure and functions of the outer retina. Dr. Jiyang Cai and colleagues are examining how RPE cell delivery bubbles (called microvesicles) can transfer genetic materials to the choroidal blood vessel cells and alter their functions. Results from this study could reveal a new cause of age-related macular degeneration (AMD). Program: Macular Degeneration  Jacque Duncan, M.D.University of California, San Francisco Title: Relationship Between Fundus Autofluorescence And Cell Survival Summary: This project will ask whether autofluorescent lesions visible in the macula and genetic risk factors associated with age-related macular degeneration (AMD) correlate with vision loss and progression of disease severity. We will use multiple measures of retinal structure and function to measure disease severity and progression over 1 year. We will correlate these precise measures of AMD disease severity and progression with autofluorescent lesions and genes associated with increased risk of AMD. Program: Macular Degeneration  Noriko Esumi, M.D., Ph.D.Johns Hopkins University Title: Mouse Models For Studying The Role Of Inflammation in AMD Summary: Age-related macular degeneration (AMD) is thought to result from abnormalities of the retinal pigment epithelium (RPE), the cell layer that nourishes and detoxifies the retina. Chronic inflammation is a major underlying condition of aging, taking its toll on many organs. Various environmental stimuli, such as oxidative stress and inflammatory molecules, converge upon a critical master regulator of inflammation. However, the specific roles of chronic inflammation and its master-regulator protein in the RPE and AMD are still unknown. Therefore, Dr. Noriko Esumi and colleagues propose to engineer two new types of mice to address the biological role of this inflammation master regulator and its key partner in the RPE, and to ultimately evaluate whether targeting these inflammation proteins is a possible strategy for AMD treatment or prevention. Program: Macular Degeneration  Krysten M. Farjo, Ph.D.University of Oklahoma Health Sciences Center Title: A Novel Visual Cycle Inhibitor to Treat Macular Degeneration Summary: Many types of macular degeneration are initiated and maintained by the accumulation of toxic vitamin A derivatives in the retina. Dr. Krysten Farjo and colleages are focusing on developing a new therapeutic agent to reduce the formation of toxic vitamin A derivatives by inhibiting vitamin A uptake into the retina. The ultimate goal is to develop a drug that could be used to slow and prevent vision loss in patients with macular degeneration. Program: Macular Degeneration  Thomas A. Ferguson, Ph.D.Washington University Title: The Role of Autophagy in Age-related Eye Disease Summary: Autophagy (self-eating) is a recycling process for our cells that continuously clears damaged structures and recycles the ingredients to maintain proper cellular function. As we get older this recycling system does not work very well and this can leads to diseases of aging such as Alzheimer's and Age-related Macular Degeneration. Our studies will determine how important this process is for vision and if we can use this information to develop a new therapy to treat blinding eye disorders. Program: Macular Degeneration  Jonathan L. Haines, Ph.D.Vanderbilt University Medical Center Title: The Genetics of AMD in African-Americans Co-PI(s):
Anita Agarwal, M.D.
Vanderbilt University Summary: The primary goal of this project is ascertain at least 100 African-American individuals with age-related macular degeneration and 100 African-American unrelated controls. We will use multiple modalities to identify and enroll these individuals. We will then test this dataset for known genetic risk factors to determine if these effects generalize in the African-American population. Program: Macular Degeneration Peter Humphries, Ph.D.Trinity College Dublin Title: The Inflammasome and Novel Therapeutic Targets in AMD Co-PI(s):
Matthew Campbell, B.Sc. (Hons), Ph.D.
Trinity College Dublin Summary: Our proposed research will aim to identify and implicate a novel inflammatory pathway in the progression of AMD. This pathway is termed the "inflammasome" and the identification of novel therapeutic targets for AMD could pave the way for more robust and reliable therapies for both dry and wet AMD. Indeed, if the inflammasome were to be implicated in the development of AMD, the targets for potential therapies would be greatly improved. Moreover, low molecular weight inhibitors or indeed augmenters of inflammasome component activation could also be investigated for therapeutic use. Program: Macular Degeneration David R. Hyde, Ph.D.University of Notre Dame Title: Generating a Zebrafish Model to Study AMD Summary: This project will address if the loss of retinal pigmented epithelial (RPE) cells is the primary or secondary cause of cone cell loss in macular degeneration. Using zebrafish, which possesses the ability to regenerate photoreceptor cells in the eye, we will also examine if the damaged RPE cells can be regenerated and the subsequent consequences on cone photoreceptor cell regeneration. Program: Macular Degeneration
Last Review: 04/29/13 |
BrightFocus Foundation |
