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Grant Funding for Macular Degeneration Research
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Macular Degeneration Research - Current Awards

Dr. Martin-Paul Agbaga

Martin-Paul Agbaga, Ph.D.

University of Oklahoma Health Sciences Center
Oklahoma City, OK

Title: What Triggers Mutant ELOVL4 Induced Blindness in STGD3?
Non-Technical Title: What is the Cause of Macular Degeneration in STGD3 Patients?
Duration: July 1, 2013 - June 30, 2015

Summary: Both Age-Related Macular Degeneration (AMD) and juvenile autosomal dominant Stargardt macular dystrophy (STGD3) share similar clinical features that result in irreversible blindness. The only difference is that, while AMD blindness starts in people over 60 years of age, STGD3 blindness starts in their teenage years. Dr. Agbaga and his team reasoned that since AMD and STGD3 share similar clinical features, there must be a common mechanism that contributes to the death of photoreceptor cells leading to blindness in both types of patients and that identification of this mechanism will help us to develop better drugs for treating these patients. The long-term goal of Dr. Agbaga’s studies is to use animal models of STGD3 to hunt and identify what sets off the death of the photoreceptor cells in these patients, with the goal that once they find the culprit, they can design effective drugs to silence it and preserve vision in STGD3 and some AMD patients.
More details

Program: Macular Degeneration
Award Type: Standard
$120,000

Acknowledgements: Recipient of the Elizabeth Anderson Award for Macular Degeneration Research



Dr. Ashery-Padan

Ruth Ashery-Padan, Ph.D.

Tel Aviv University
Israel

Title: Roles for RPE-specific MicroRNAs in Age-related Retinal Diseases
Non-Technical Title: Involvement of microRNAs in Retinal Diseases
Duration: July 1, 2013 - June 30, 2015

Co-PI(s):
Yechiel Elkabetz, Ph.D.
Tel Aviv University

Summary: Age-related macular degeneration (AMD) is a complex disease, associated with a combination of genetic and environmental factors. The early stages of AMD involve dysfunction of the retinal pigmented epithelium (RPE)— cells which are important for the function and survival of the photoreceptors. RPE play a role in processes related to adhesion (“glue” that holds cells together), protection against oxidative damage, triggering of the immune response, and regulation of new blood vessel growth of the choriocapillaris (vessels in the back of the eye). MicroRNAs (miRNAs) are a large group of small RNA molecules that have been recently identified to simultaneously inhibit the expression of many genes. Thus, miRNAs are implicated in controlling a wide range of normal physiological and disease events. In the course of this study, Drs. Ashery-Padan’s and Elkabetz’ team will identify the involvement of miRNAs in processes that are altered in AMD. The team will employ a combination of genetic, cellular, biochemical and molecular techniques that will enable them to reveal the involvement of miRNAs in AMD and other degenerative diseases of the retina.
More details

Program: Macular Degeneration
Award Type: Standard
$120,000



Dr. Milam A. Brantley, Jr.

Milam A. Brantley, Jr., M.D., Ph.D.

Vanderbilt University Medical Center
Nashville, TN, United States

Title: Quantitative Evaluation of Environmental Risk for AMD
Non-Technical Title: Evaluation of Environmental Risk for AMD
Duration: July 1, 2012 - June 30, 2014

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.
More details

Program: Macular Degeneration
Award Type: Standard
$100,000



Dr. Jiyang Cai

Jiyang Cai, Ph.D.

University of Texas Medical Branch
Galveston, TX, United States

Title: Exosomal microRNA from the RPE
Non-Technical Title: Small RNAs as signaling molecules between the RPE and choroid
Duration: July 1, 2012 - June 30, 2014

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).
More details

Program: Macular Degeneration
Award Type: Standard
$100,000



Dr. Venkata Chavali

Venkata Chavali, Ph.D.

University of Pennsylvania School of Medicine
Philadelphia, PA

Title: Association And Functional Characterization Of Long Non-Coding RNAs (lncRNAs) In Age-related Macular Degeneration
Non-Technical Title: Investigation of the Role and Function of Long Non-Coding RNAs Which May Serve As Novel Molecular Tools that Cause Age-Related Macular Degeneration
Duration: July 1, 2013 - June 30, 2015

Summary:

The function of long non-coding RNA (lncRNA) in the genome and their role in biological processes and disease is not well studied even though some lncRNA are known to be associated with neurological diseases. Dr. Chavali’s team proposes to sequence the total retina and retinal pigment epithelium (RPE) tissue transcriptome (the total collection of RNAs that are expressed within cells at that point in time) from normal and age-related macular degeneration (AMD) donor eyes. Analysis of these sequences will reveal the differentially expressed (DE) lncRNAs and messenger RNAs (mRNAs) that are relevant to AMD and expressed in the retina and RPE/choroid. The team intends to characterize the role of DE lncRNAs in AMD and understand how lncRNAs potentially influence the development of AMD. The outcome of this project will provide novel pathways to understand what is involved with the initiation and progression of AMD. Defining the expression changes of lncRNAs in AMD may lead to the development of new biomarkers and therapies.


More details

Program: Macular Degeneration
Award Type: Standard
$120,000

Acknowledgements: This grant is made possible by bequests from the Helen Juanita Reed Irrevocable Trust and the Helen Juanita Reed Charitable Remainder Unitrust. Recipient of The Helen Juanita Reed Memorial Award.



Dr. Jing Chen

Jing Chen, Ph.D.

Boston Children's Hospital
Boston, MA

Title: Control of Neovascular AMD by Nuclear Receptor RORalpha
Non-Technical Title: A New Mechanism Linking Lipid and Altered Inflammation in Neovascular AMD
Duration: July 1, 2013 - June 30, 2015

Summary: A major cause of blindness in AMD is abnormal blood vessel growth in the back of the eye (choroidal neovascularization), the “wet” form of AMD. The proposed work will help define a novel pathway linking dysregulation of lipid homeostasis (fat levels) with altered inflammatory responses in AMD. Both processes are fundamentally important to the development and progression of wet AMD. Dr. Chen’s discoveries may help to develop a new way to treat AMD by specifically changing the levels of a lipid sensing nuclear receptor protein that modifies inflammation.
More details

Program: Macular Degeneration
Award Type: Standard
$120,000



Dr. Sarah L. Doyle

Sarah L. Doyle, B.A., Ph.D.

Trinity College Dublin
Dublin, Ireland

Title: Investigating a Role for Toll like Receptor 2 Signaling in the Pathogenesis of AMD
Non-Technical Title: Investigating if an Uncontrolled Immune Response to Your Own Damaged Cells Causes the Progression of AMD
Duration: July 1, 2013 - June 30, 2015

Co-PI(s):
Matthew Campbell, Ph.D.
Trinity College Dublin
Luke O'Neill, Ph.D.
Trinity College Dublin

Summary: The first clinical sign that an individual has age-related macular degeneration (AMD) is the presence of yellowish deposits, which accumulate in the area at the back of the eye known as the macula. The macula is responsible for your central vision; holding two coins in front of your eyes results in a single large black circle blocking your central vision, which is a realistic simulation of what it is like to live with advanced AMD. Typically, inflammatory responses of the immune system are caused as a result of microbial infection. However, in chronic conditions, a form of “sterile” inflammation can exist in areas of the body in response to damaged “self-elements” in the absence of infection, due to uncontrolled activation of immune system sensors. Drs. Doyle and Campbell have found that this “sterile” inflammation is central to the progression of AMD, and they will investigate the pathways involved with this process, potentially leading to new treatments for AMD.
More details

Program: Macular Degeneration
Award Type: Standard
$120,000



Dr. Noriko Esumi

Noriko Esumi, M.D., Ph.D.

Johns Hopkins University
Baltimore, MD, United States

Title: Mouse Models For Studying The Role Of Inflammation in AMD
Non-Technical Title: Mouse Models For Studying Factors That Control Inflammation in AMD
Duration: July 1, 2012 - June 30, 2014

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.
More details

Program: Macular Degeneration
Award Type: Standard
$100,000



Dr. Krysten M. Farjo

Krysten M. Farjo, Ph.D.

University of Oklahoma Health Sciences Center
Oklahoma City, OK, United States

Title: A Novel Visual Cycle Inhibitor to Treat Macular Degeneration
Non-Technical Title: Developing a New Therapeutic for Macular Degeneration
Duration: July 1, 2012 - June 30, 2014

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.
More details

Program: Macular Degeneration
Award Type: Standard
$100,000



Dr. Chenghua Gu

Chenghua Gu, D.V.M., Ph.D.

Harvard Medical School
Boston, MA

Title: The Role of Semaphorin 3E Signaling in Neovascular AMD
Non-Technical Title: New Therapeutic Strategy for Neovascular AMD
Duration: July 1, 2013 - June 30, 2015

Summary: Patients with neovascular age-related macular degeneration (wet AMD) have benefited from therapies that inhibit the formation of new blood vessels in the retina. These medications on the market, including pegaptanib (Macugen®), ranibizumab (Lucentis®), bevacizumab (Avastin®), and EYLEA®, directly bind VEGF, a protein that stimulates blood vessel formation in the eye. Dr. Gu’s team proposes to improve the therapies for wet AMD by finding novel therapies that could be used alone or in combination with the existing therapeutics. Dr. Gu’s laboratory has recently identified a new signaling pathway through the proteins Semaphorin 3E (Sema3E) and Plexin-D1 that inhibits the function of VEGF. More importantly, this pathway only functions in the newly formed abnormal blood vessels, but not in the normal blood vessels. Thus, through searching for new peptides and drugs that target this pathway, the team will discover novel therapeutic agents that expand the repertoire in fighting AMD and improve the quality of life for patients suffering from AMD.
More details

Program: Macular Degeneration
Award Type: Standard
$120,000

Acknowledgements: This grant is made possible by a bequest from the Rasmussen Family Trust.



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Last Review: 08/30/13