This project seeks to determine whether dampening inflammation using a newly developed strategy can prevent a prevalent, currently untreatable inherited eye disease, Stargardt disease. Our approach is unique in that it can be ‘turned on’ or ‘turned off’ in the eye when necessary, thereby minimizing potential detrimental off-target effects associated with current similar strategies. We anticipate that this unique aspect of our strategy will make it more likely to be effectively used ultimately in humans with Stargardt disease.
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- John Hulleman, PhDThe University of Texas Southwestern Medical Center (Dallas, TX)ID:M2018099October 1, 2018 to September 30, 2020Macular DegenerationStandard$160,000
- Brett Collins, PhDThe University of Queensland (Brisbane, Queensland, Australia)
With our aging population comes an ever-increasing incidence of Alzheimer’s disease (AD), and other age-related dementias. By the year 2050, there is forecast to be more than 13 million people living with AD in the USA, and new treatments are desperately needed to prevent this impending epidemic. There are currently no effective therapies for AD, with current clinical trials all attempting to directly target amyloid beta (Aβ) peptides thought to be the driver of neuronal degeneration. In the long term, scientists believe that we may have to use cocktails of drugs to effectively slow the disease, much as we now do for diseases such as HIV/AIDS. A new concept in AD research is that cellular processes regulating protein turnover (ie, the balance between protein synthesis and protein degradation) could be manipulated to prevent the build-up of the toxic Aβ peptides that cause neurological failure. In this work, we will be developing novel small molecules and peptides that we hope will enhance this protein turnover in neurons, and provide a starting point for designing new AD drugs.ID:A2018627SJuly 18, 2018Alzheimer's DiseaseStandard$191,034
- Charles Glabe, PhDUniversity of California, Irvine (Irvine, California)
In the continuously aging, modern population of developing countries, understanding of the mechanisms that contribute to the development to age-related, neurodegenerative diseases is of the utmost importance. Disturbances in cellular regulation, e.g., on the level of the signaling of cell death might significantly contribute to the occurrence, propagation and severity of pathophysiological states such as the occurrence and development of Alzheimer’s disease. Especially, cell death pathways that are involved in the progression of the inflammatory response, one of the hallmarks of AD, are of highest interest. Importantly, detailed knowledge about this specific type of inflammatory cell death pathway, its spatial and temporal distribution in AD brains might allow us to identify potential therapeutic strategies to prevent neurodegeneration.
In the continuously aging, modern population of developing countries, understanding of the mechanisms that contribute to the development to age-related, neurodegenerative diseases is of the utmost importance. Disturbances in cellular regulation, e.g., on the level of the signaling of cell death might significantly contribute to the occurrence, propagation and severity of pathophysiological states such as the occurrence and development of Alzheimer’s disease. Especially, cell death pathways that are involved in the progression of the inflammatory response, one of the hallmarks of AD, are of highest interest. Importantly, detailed knowledge about this specific type of inflammatory cell death pathway, its spatial and temporal distribution in AD brains might allow us to identify potential therapeutic strategies to prevent neurodegeneration.ID:A2018718SJuly 1, 2018 to June 30, 2021Alzheimer's DiseaseStandard$300,000
- Karl Wahlin, PhDUniversity of California, San Diego (La Jolla, CA)
Macular degenerative disease affects millions worldwide and models to study the condition in humans are generally lacking. We have developed human disease-based stem cell lines (created from adult stem cells) that can be readily converted into retinal pigment epithelium (RPE) in order to study the disease process in the laboratory. Unique to this project, we have also designed a fluorescent protein reporter that will allow us to study the temporal dynamics of RPE cell dystrophy, thus allowing the systematic optimization of drug screening aimed at reducing protein deposits typical of AMD.ID:M2018175July 1, 2018 to June 30, 2020Macular DegenerationStandard$160,000
Recipient of the Carolyn K. McGillvray Award for Macular Degeneration Research
- Monica Jablonski, PhDThe University of Tennessee Health Science Center (Memphis, TN)
Millions of people are affected by glaucoma and some lose their vision due to this disease. To develop new drugs to treat glaucoma or to understand why glaucoma causes vision loss, it is important to have accurate models of the disease. Unfortunately, there are not enough models available that truly reflect the human disease. We hope to change that. In our study, we will identify and characterize new glaucoma models that share the disease phenotypes of humans. These models will be a very useful resource for all vision scientists.ID:G2018116July 1, 2018 to June 30, 2020GlaucomaStandard$150,000
- Daniel Geschwind, MD, PhDUniversity of California, Los Angeles (Los Angeles, CA)
Recent scientific discoveries suggest that multiple cell types might participate in Alzheimer’s disease (AD), and understanding the key players and their effects on dementia would advance our ability to design new drugs and therapies. However, the complexity of the brain’s different cell types presents a unique challenge to scientific inquiry. Here I propose work to bridge the divide by using cutting edge technology to profile the different cells of the dementia brain at unprecedented resolution. The results of this work will be new candidate drug targets for dementia and a new approach for studying complex brain diseases.ID:A2018700SCo-principal Investigators:Jessica Rexach, MD, PhDJuly 1, 2018 to June 30, 2021Alzheimer's DiseaseStandard$300,000
- Goonho Park, PhDUniversity of California, San Diego (La Jolla, CA)
Neurons communicate one another by forming synapses in brain. For reasons that are not completely clear, these synaptic connections are susceptible to damage and are lost in the early stages of AD. Injury and loss of synapses in the brain are believed to be a major reason for cognitive impairment seen in individuals with AD. Amyloid-beta peptide (Aβ), which is generated from amyloid precursor protein (APP), is hypothesized to be one of the major reasons for synaptic damage. In addition, however, APP also generates another fragment called C31 which we hypothesize could play an additional role in synaptic injury. In this project, we will test whether blocking C31’s generation from APP can protect synapses from injury and damage.ID:A2018212FJuly 1, 2018 to June 30, 2020Alzheimer's DiseasePostdoctoral Fellowship$150,000
- Stephanie Rainey-Smith, PhDEdith Cowan University (Perth, Australia)
This study will explore the relationship between sleep, memory and thinking, and changes in the brain in Alzheimer’s disease (AD) by investigating whether improved sleep (better and longer) causes better memory and thinking, slower protein build up in the brain and slows the shrinking of the brain. The results will help us find a way to slow or stop this horrible disease. With sleep problems reported in 60 percent of adults over 65, my research will impact a significant proportion of the population.ID:A2018402FCollaborators:Melissa Ree, PhD; Peter Eastwood, PhD; Hamid R. Sohrabi, PhD; Victor L. Villemagne, MDMentors:Ralph N. Martins, PhDJuly 1, 2018 to June 30, 2020Alzheimer's DiseasePostdoctoral Fellowship$149,998
- Yang Hu, MD, PhDStanford University (Stanford, CA)
Glaucoma is the most common cause of irreversible blindness and by 2040 will affect more than 100 million people between 40 and 80 years of age, worldwide. Glaucoma is characterized by optic nerve neuropathy with retinal ganglion cell (RGC) axon degeneration followed by progressive RGC death. Understanding gene regulation mechanisms that are associated with RGCs at normal function, under disease, or after treatment, is essential for identifying novel therapeutic targets and innovative and efficient neural repair strategies. We are taking advantage of newly developed genetic tools to elucidate the comprehensive gene regulatory networks that will serve as a blueprint for developing novel and effective neuroprotectants for glaucoma.ID:G2018183July 1, 2018 to June 30, 2020GlaucomaStandard$150,000
- Jason Porter, PhDUniversity of Houston (Houston, TX)
Glaucoma is the leading cause of irreversible blindness worldwide (estimated to affect over 60 million people) and is generally considered to be caused by the damage of retinal ganglion cell (RGC) axons and the death of RGCs. Previous studies support the idea that the loss of radial peripapillary capillaries may play an important role in axonal degeneration in glaucoma. This project will use high-resolution in vivo imaging to better clarify changes in the radial peripapillary capillaries and optic nerve head in relation to neuronal damage in living eyes with experimental glaucoma. The results of the proposed work may aid in earlier diagnosis and management of this disease by providing an earlier structural marker for detecting glaucomatous damage compared with current clinical measures.ID:G2018061Collaborators:Nimesh Patel, OD, PhD; Hope Queener, MSJuly 1, 2018 to June 30, 2020GlaucomaStandard$150,000