Grant List
Represents Grant table in the DB
GET /v1/grants?page%5Bnumber%5D=1405&sort=end_date
{ "links": { "first": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1&sort=end_date", "last": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1405&sort=end_date", "next": null, "prev": "https://cic-apps.datascience.columbia.edu/v1/grants?page%5Bnumber%5D=1404&sort=end_date" }, "data": [ { "type": "Grant", "id": "14694", "attributes": { "award_id": "1R35HL171346-01", "title": "Transcriptional and epigenetic mechanisms of alveologenesis and re-alveologenesis", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Heart Lung and Blood Institute (NHLBI)" ], "program_reference_codes": [], "program_officials": [ { "id": 23738, "first_name": "Sara", "last_name": "Lin", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-03-15", "end_date": "2031-02-28", "award_amount": 1122351, "principal_investigator": { "id": 31389, "first_name": "Jichao", "last_name": "Chen", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [], "awardee_organization": { "id": 897, "ror": "", "name": "CINCINNATI CHILDRENS HOSP MED CTR", "address": "", "city": "", "state": "OH", "zip": "", "country": "United States", "approved": true }, "abstract": "Lung diseases impacting the gas exchange alveoli, including COVID-19, are becoming the leading cause of death. A multi-lineage, transcriptional, and epigenetic understanding of alveologenesis and re- alveologenesis upon injury is a timely response to the disease burden and leverages latest single-cell technology. My lab’s track record in studying the lung epithelial, endothelial, and mesenchymal lineages lays the foundation for pursuing a poorly understood process of cellular maturation (Theme 1), a recently identified capillary cell type (Theme 2), and a novel signaling regulation of distinct mesenchymal cell populations (Theme 3). The anticipated knowledge will tackle fundamental questions of cell fate, plasticity, and signaling; shed light on bronchopulmonary dysplasia, pulmonary hypertension, acute lung injury, as well as non-coding variants from genome-wide association studies; and opens the door to single-cell functional genomics applicable to any organ.", "keywords": [ "Acute Lung Injury", "Alveolus", "Bioinformatics", "Blood capillaries", "Bronchopulmonary Dysplasia", "COVID-19", "Cause of Death", "Cell Lineage", "Cell Physiology", "Cells", "Development", "Endothelium", "Epigenetic Process", "Epithelium", "Foundations", "Gases", "Genetic Transcription", "Injury", "Knowledge", "Lung", "Lung Diseases", "Mesenchymal", "Organ", "Population", "Pulmonary Hypertension", "Reaction Time", "Regulation", "Signal Transduction", "Untranslated RNA", "Variant", "burden of illness", "cell type", "functional genomics", "genome wide association study", "mouse genetics", "novel", "single cell genomics", "single cell technology" ], "approved": true } }, { "type": "Grant", "id": "14592", "attributes": { "award_id": "1UM1TR004771-01", "title": "CTSA UM1 at the University of Alabama at Birmingham", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Center for Advancing Translational Sciences (NCATS)" ], "program_reference_codes": [], "program_officials": [ { "id": 21618, "first_name": "Audie A", "last_name": "Atienza", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-05-01", "end_date": "2031-04-30", "award_amount": 9869052, "principal_investigator": { "id": 31258, "first_name": "PATRICE", "last_name": "DELAFONTAINE", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 11466, "first_name": "Robert P.", "last_name": "Kimberly", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 612, "ror": "https://ror.org/008s83205", "name": "University of Alabama at Birmingham", "address": "", "city": "", "state": "AL", "zip": "", "country": "United States", "approved": true } ] }, { "id": 31259, "first_name": "Orlando M", "last_name": "Gutierrez", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 612, "ror": "https://ror.org/008s83205", "name": "University of Alabama at Birmingham", "address": "", "city": "", "state": "AL", "zip": "", "country": "United States", "approved": true }, "abstract": "– The Center for Clinical and Translational Science (CCTS), the CTSA Hub at the University of Alabama at Birmingham (UAB), serves a region of the country with a disproportionate burden of multiple chronic diseases and health disparities. The COVID-19 pandemic brought into sharp relief the critical importance of translating new scientific discoveries into interventions that improve the health of patients and their communities in an efficient, effective and equitable manner. The unique health challenges faced by our communities previously prompted the creation of the CCTS Partner Network – spanning Alabama, Mississippi and Louisiana – to ensure that our research and training efforts serve the populations in our region while maximizing collaborative synergies in clinical and translational science (CTS) investigation to catalyze discovery and accelerate the dissemination and implementation of evidence toward health impact. As it continues to serve as a national resource for responding to public health emergencies, the CCTS Partner Network will provide programmatic leadership and shared governance (Aim 1) to mobilize the resources and talents throughout the region as it brings together academic, health system, industry and community partners to advance discovery science in concert with the CTSA consortium. The CCTS will further the development of a vibrant, diverse clinical and translational research workforce (Aim 2) by expanding programs that provide both didactic and experiential training to convey new skills, perspectives and understanding of the translation process for faculty, trainees, clinical research professionals and community alike. The Center will promote community and stakeholder engagement (Aim 3) in trusting, bidirectional relationships in all aspects of the research process to develop, demonstrate, disseminate and implement new discoveries to enhance the impact of health insights on those who will most benefit. It will leverage expertise in health informatics, clinical research informatics and translational bioinformatics to extend collaborative, coordinated data analytics and digital innovations across the Partner Network and the CTSA consortium (Aim 4) that allow full utilization of real world data together with rich, deep clinical information to enable discovery research from the bench to the learning healthcare system. The CCTS Hub and Network will support ethical, scientifically rigorous, informative clinical trials and pilot studies by providing a range of specialized services, resources and consultations (Aim 5) guided by NCATS principles of effective translational science. Through these efforts, the CCTS will formalize its CTS Research Program (Aim 6) to identify, develop and test novel approaches to overcome significant roadblocks in biomedical research, generating new insights that can be generalized to other CTSA Hubs. By achieving these aims, the CCTS will harness the Network’s vibrant collaborative energy to accelerate the discovery, dissemination and implementation of new findings, deliver treatments to more people more quickly, reduce the burden of chronic disease and advance health equity in the Deep South and beyond.", "keywords": [ "Acceleration", "Address", "Affect", "Alabama", "Area", "Back", "Bioinformatics", "Biomedical Research", "Biotechnology", "COVID-19 pandemic", "Chronic Disease", "Clinical", "Clinical Research", "Clinical Sciences", "Clinical Trials", "Communication", "Communities", "Complement", "Consultations", "Country", "Data", "Data Analytics", "Dedications", "Deep South", "Development", "Disease", "Disparity", "Dissemination and Implementation", "Diverse Workforce", "Ecosystem", "Ensure", "Equity", "Ethics", "Evaluation", "Faculty", "Feedback", "Future", "Goals", "Growth", "Health", "Health Sciences", "Health system", "Healthcare Systems", "Informatics", "Infrastructure", "Institution", "Intervention", "Investigation", "Knowledge", "Leadership", "Learning", "Louisiana", "Malignant Neoplasms", "Medical center", "Mentors", "Mission", "Mississippi", "National Center for Advancing Translational Sciences", "Neurologic", "Patients", "Persons", "Pilot Projects", "Population", "Process", "Public Health", "Public Health Informatics", "Research", "Research Methodology", "Research Project Grants", "Research Support", "Resource Informatics", "Resources", "Science", "Scientific Advances and Accomplishments", "Services", "Special needs", "Talents", "Testing", "Training", "Training Programs", "Training and Education", "Translating", "Translation Process", "Translational Research", "Trust", "Universities", "Vision", "Work", "burden of chronic illness", "cardiometabolism", "career", "clinical center", "community partners", "community partnership", "data interoperability", "design", "digital", "disease disparity", "evidence base", "health care delivery", "health disparity", "health equity", "high dimensionality", "improved", "industry partner", "innovation", "insight", "multidisciplinary", "novel strategies", "novel therapeutics", "programs", "public health emergency", "research study", "skills", "synergism", "tool" ], "approved": true } }, { "type": "Grant", "id": "15386", "attributes": { "award_id": "1UM1TR004772-01", "title": "Dartmouth Clinical and Translational Science Institute", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Center for Advancing Translational Sciences (NCATS)" ], "program_reference_codes": [], "program_officials": [ { "id": 21567, "first_name": "Jamie Mihoko", "last_name": "Doyle", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-07-25", "end_date": "2031-06-30", "award_amount": 4073191, "principal_investigator": { "id": 31986, "first_name": "Steven L", "last_name": "Bernstein", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, "other_investigators": [ { "id": 31987, "first_name": "KEITH D.", "last_name": "PAULSEN", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 31988, "first_name": "Anna N. A.", "last_name": "Tosteson", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 2072, "ror": "", "name": "DARTMOUTH-HITCHCOCK CLINIC", "address": "", "city": "", "state": "NH", "zip": "", "country": "United States", "approved": true }, "abstract": "The COVID-19 pandemic brought into stark relief the many healthcare challenges facing individuals living in rural areas—the compounded needs of an aging population, geographic dispersion, inadequate public transportation, spotty internet service, spikes in substance use, vaccine hesitancy. These disparities—like most public health problems—reflect a complex interplay of biological, environmental, psychological, social, and systems-level factors. At the same time, the pandemic highlighted potential solutions and spurred a wealth of research in healthcare delivery science in rural areas. Much of this work was conducted by scientists affiliated with SYNERGY, Dartmouth’s Clinical and Translational Science Institute, founded in 2013. SYNERGY faculty reported the expanded use of telehealth, digital health, machine learning; strengthened partnerships between healthcare systems and community health centers; and developed a “COVID compass” to guide policymaking. Our work in this area, centered in SYNERGY’s earliest years, has deepened our commitment to translational science that centers rural healthcare delivery and health equity, while exploring the full spectrum of translational science and workforce development. Hence, we propose to return SYNERGY to the national CTSA consortium. The overarching goal of this application is to continue to spur innovation in clinical and translational science with a focus on rural healthcare, in collaboration with other CTSA hubs, and to study a new model to catalyze T3 translational science in healthcare settings. SYNERGY reflects a close partnership between Dartmouth Health (DH) and Dartmouth College (DC). DH is the largest healthcare system in New Hampshire, with its flagship hospital in a rurally designated area. DC brings the resources of a research- intensive college, including schools of medicine, engineering, business, and graduate studies. SYNERGY includes our new Center for Rural Healthcare Delivery Science and key regional collaborators, including the Northern New England Clinical Translational Research Network (a partnership between MaineHealth and the University of Vermont), a Veterans Affairs hospital, community groups, and a “pipeline” program to grow the scientific workforce. SYNERGY’s goals are to (1) Accelerate the delivery of evidence-based diagnostics, therapeutics, and processes to address the healthcare needs of rural communities; (2) Assure the availability of timely, actionable patient- and population-level data to mitigate the translational block of “siloing” between translational scientists and healthcare system leadership by deploying a novel coproduction learning health system (LHS); (3) Train the next generation of translational scientists, with a particular focus on identifying future leaders in healthcare delivery science and rural health; (4) Disseminate these practices and lessons learned within the CTSA community through engagement with subnetworks addressing rural health, implementation science, and learning health systems science; and (5) Involve local communities in the design, conduct, analysis, and dissemination of our work, while engaging in studies of stakeholder engagement.", "keywords": [], "approved": true } }, { "type": "Grant", "id": "15401", "attributes": { "award_id": "1UM1TR004906-01", "title": "Center for Clinical and Translational Sciences", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Center for Advancing Translational Sciences (NCATS)" ], "program_reference_codes": [], "program_officials": [ { "id": 12440, "first_name": "CAROL", "last_name": "MERCHANT", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-07-24", "end_date": "2031-06-30", "award_amount": 5707763, "principal_investigator": { "id": 24633, "first_name": "Maria Eulalia", "last_name": "Fernandez", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 788, "ror": "", "name": "UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON", "address": "", "city": "", "state": "TX", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [ { "id": 12442, "first_name": "David D", "last_name": "McPherson", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 24637, "first_name": "Belinda", "last_name": "Reininger", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 32000, "first_name": "LORNA H.", "last_name": "MCNEILL", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 32001, "first_name": "FUNDA", "last_name": "MERIC-BERNSTAM", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] }, { "id": 32002, "first_name": "JIAJIE", "last_name": "ZHANG", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "awardee_organization": { "id": 788, "ror": "", "name": "UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON", "address": "", "city": "", "state": "TX", "zip": "", "country": "United States", "approved": true }, "abstract": "PROJECT SUMMARY/ ABSTRACT Since its inception in 2006, our CCTS has assembled a well-integrated, and high-impact network in Texas including 6 academic institutions: The University of Texas Health Science Center at Houston (UTH-H), the University of Texas M.D. Anderson Cancer Center (MDACC), Rice University, University of Texas at Tyler (UT- Tyler), University of Texas Rio Grande Valley (UT-RGV), and two UTH-H affiliated hospital systems (Memorial Hermann and Lyndon B. Johnson County Hospital). We have actively supported and integrated our partners into NCATS national initiatives, including recent recruitment of a large number of diverse patients for COVID-19 trials and studies. Over the past 17 years, our CCTS has supported investigators at 30 institutions from over 97 biomedical fields who disseminate scientific and healthcare initiatives that impact diverse populations. By expanding our network to include Texas Tech University Health Sciences Center El Paso (TT-EP), our catchment now encompasses ~23,000 sq. miles and over 16 million residents (4% of the U.S. population) showing some of the greatest diversity in the country. Each partner institution brings communities with unique geographic, demographic, cultural and socio-economic characteristics and challenges. In the next cycle, we will thoroughly integrate translational science into our entire program and expedite the output of scientific observations policies discoveries, clinical and community experiences into effective treatments, clinical interventions and public health that improve the health of people within our diverse state and beyond.Our shared leadership model and partnerships across the disparate regions of Texas will enable us to carry out our 6 strategic goals: (1) Enhance our robust organizational structure of Cores to improve and expand the impact of CCTS programs on translational science (Element B); (2) Promote partnerships and collaborations of stakeholders that extend benefits of translational science to underserved populations (Element B & Module C2); (3) Enhance integration of D&I sciences across our CTSA (Module C2); (4) Innovate scientific and operational functions of CRUs to expand the effectiveness and accessibility of CTS in broadly heterogeneous populations (Module D1); (5) Foster education and career training in translational science by establishing methods that inspire our growing workforce of clinical research professionals (Modules C1 & D2); (6) Create and disseminate innovative Health Informatics (HI) solutions to become a national resource for the response to urgent public health needs of underserved populations (Module D3). To accomplish these strategic goals, we will pursue our 3C's and 5I's motto: Community, Collaboration & Careers (3C's) and Impact, Inclusion, Implementation, Innovation, & Informatics (5I's). Our leadership and organizational model, resources, partnerships, and research infrastructure have been strategically planned so diagnosis, goal that more people get the benefits of translational science, from access to accurate efficacious treatment to cost-effective disease prevention strategies. This is our mission and our major which reflectsNCATS's vision: a world with “more treatments for ALL people more quickly”.", "keywords": [], "approved": true } }, { "type": "Grant", "id": "15530", "attributes": { "award_id": "1R35NS137480-01", "title": "Epitranscriptomic regulation in the mammalian nervous system", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Neurological Disorders and Stroke (NINDS)" ], "program_reference_codes": [], "program_officials": [ { "id": 22867, "first_name": "TIMOTHY M", "last_name": "LAVAUTE", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-12-23", "end_date": "2032-11-30", "award_amount": 985026, "principal_investigator": { "id": 12608, "first_name": "Guo-li", "last_name": "Ming", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 232, "ror": "https://ror.org/00b30xv10", "name": "University of Pennsylvania", "address": "", "city": "", "state": "PA", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [], "awardee_organization": { "id": 232, "ror": "https://ror.org/00b30xv10", "name": "University of Pennsylvania", "address": "", "city": "", "state": "PA", "zip": "", "country": "United States", "approved": true }, "abstract": "Epitranscriptomics, analogous to the epigenetic code formed by DNA and histone modifications, is the study of more than 170 chemically distinct types of RNA modifications, which modulate nearly all aspects of RNA metabolism, such as splicing, translocation, decay, stability, and translation. The recent profound success of COVID19 mRNA vaccines utilizing the pseudo-uridine modification highlights the translational potential of epitranscriptomics. Emerging evidence suggests diverse roles and mechanisms of dynamic RNA modifications in the mammalian nervous system and the association of epitranscriptomic dysregulations with developmental, neurological, psychiatric, and degenerative brain disorders. The majority of recent epitranscriptomic studies used cultured immortalized cell lines and the physiological functions of various RNA modifications remain largely unexplored. Recent technical advances in human induced pluripotent stem cell (iPSC)-derived brain organoids and genome editing open doors to investigate epitranscriptomic regulation in human brain development processes and associated brain disorders. The overarching goal of this research program is to investigate roles and mechanisms of epitranscriptomic regulation in the development and function of the mammalian nervous system, and pathological consequences of disrupting these processes, using both mouse and human iPSC-derived 2D and 3D brain organoid models. There are three interrelated projects designed to test innovative hypotheses and generate foundational data for the field. In Project 1, we will focus on the development of the hypothalamus, an understudied brain region that regulates many key physiological functions, such as sleep, reproduction, and feeding, through its distinct nuclei. Based on our preliminary finding of adult-onset obesity of mice with defective m6A signaling, we will test the hypothesis that m6A signaling regulates the fate specification of neural stem cells in the arcuate nucleus for generating feeding-related neurons both in mice and human arcuate organoids. In Project 2, we will use novel sequencing technology to reveal the landscape of locally translated transcripts at synapses and investigate the role of m6A signaling in regulating activity-dependent local translation of these transcripts at synapses in the mouse hippocampus and human hippocampal organoids. In Project 3, we will focus on several risk genes associated with microcephaly that encode writer proteins for diverse epitranscriptomic modifications beyond m6A. We will generate isogenic iPSC lines and genetically modified animal models to test the functional roles and mechanisms of these RNA modifications in cortical neurogenesis. Together, we will use several orthogonal approaches to investigate functional roles and mechanisms of neuroepitranscriptomics in regulating the mammalian nervous system and its causal roles in mediating some forms of developmental pathology. The research program will also provide a platform to train the next generation of scientists from diverse backgrounds at different career stages.", "keywords": [ "3-Dimensional", "Adult", "Animal Model", "Brain", "Brain Diseases", "Brain region", "COVID-19", "Cell Line", "Cell Nucleus", "Chemicals", "Code", "DNA Modification Process", "Data", "Development", "Epigenetic Process", "Genetically Modified Animals", "Goals", "Hippocampus", "Human", "Hypothalamic structure", "Mediating", "Microcephaly", "Modeling", "Modification", "Mus", "Nervous System", "Neurologic", "Neurons", "Obese Mice", "Organoids", "Pathologic", "Pathology", "Physiological", "Process", "Proteins", "Pseudouridine", "RNA", "RNA Splicing", "RNA metabolism", "RNA vaccine", "Regulation", "Reproduction", "Research", "Role", "Scientist", "Signal Transduction", "Sleep", "Specific qualifier value", "Structure of nucleus infundibularis hypothalami", "Synapses", "Testing", "Training", "Transcript", "Translating", "Translations", "career", "cell immortalization", "design", "epitranscriptomics", "feeding", "genome editing", "histone modification", "human induced pluripotent stem cells", "induced pluripotent stem cell", "innovation", "nerve stem cell", "neurogenesis", "next generation", "novel sequencing technology", "programs", "risk variant", "success", "translational potential" ], "approved": true } }, { "type": "Grant", "id": "15531", "attributes": { "award_id": "1R35NS137447-01", "title": "Expanding insights into FTD disease mechanisms", "funder": { "id": 4, "ror": "https://ror.org/01cwqze88", "name": "National Institutes of Health", "approved": true }, "funder_divisions": [ "National Institute of Neurological Disorders and Stroke (NINDS)" ], "program_reference_codes": [], "program_officials": [ { "id": 23658, "first_name": "FRANK PAUL", "last_name": "Shewmaker", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [] } ], "start_date": "2024-12-15", "end_date": "2032-11-30", "award_amount": 1138538, "principal_investigator": { "id": 20546, "first_name": "LEONARD", "last_name": "PETRUCELLI", "orcid": null, "emails": "", "private_emails": "", "keywords": null, "approved": true, "websites": null, "desired_collaboration": null, "comments": null, "affiliations": [ { "id": 1414, "ror": "", "name": "MAYO CLINIC JACKSONVILLE", "address": "", "city": "", "state": "FL", "zip": "", "country": "United States", "approved": true } ] }, "other_investigators": [], "awardee_organization": { "id": 1414, "ror": "", "name": "MAYO CLINIC JACKSONVILLE", "address": "", "city": "", "state": "FL", "zip": "", "country": "United States", "approved": true }, "abstract": "Frontotemporal lobar degeneration (FTLD), which underlies frontotemporal dementia (FTD), encompasses a group of disorders with significant genetic, clinical, and neuropathological heterogeneity. FTLD is also genetically and pathologically associated with the motor neuron disease amyotrophic lateral sclerosis (ALS), with some patients developing both disorders. Understanding the diverse mechanisms governing FTLD pathogenesis is a fundamental area of interest of my research program, and we pursue this goal by asking impactful questions and applying innovative techniques. To accelerate scientific discovery, we have adopted a comprehensive approach that investigates multiple FTLD mechanisms driven by key molecular players like C9orf72, TDP-43, progranulin, tau and, more recently, TMEM106B. We also place great emphasis on translational research geared towards identifying much needed biomarkers and therapies, an area of particular importance given that there exists no treatment for FTLD. Since the funding of my current R35 at the end of 2016, my group has uncovered seminal findings related to the pathomechanisms mediated by FTLD-associated mutations in C9orf72 and GRN and shed crucial insight into the consequences of pathogenic TDP-43 and tau deposition in the brain. These findings have garnered high-impact publications in Science, Nature, and Cell and inspired new and ongoing avenues of research in my lab. The flexibility afforded by the R35 funding opportunity also allowed us to branch into other related topics and tackle urgent issues in the broader neuroscience field, including the need for biomarkers and mouse models for distinct repeat-associated disorders like spinocerebellar ataxias and the recent pressing need for tools to study and understand COVID-19 and its impact on the brain. Our productivity is influenced by the excellent research environment fostered at Mayo Clinic, which brings together highly interactive and devoted neurobiologists, geneticists, neuropathologists and physician scientists, the diversity of my team, and the numerous collaborations we have forged with world-renowned experts in the field, as well as our dedication to stewardship and the sharing of information and resources with the scientific community at large. Drawing from our past work on FTLD, we now propose to explore current cutting-edge questions related to: (1) the molecular underpinnings of TDP-43 localization and function and the downstream consequences of its dysfunction in disease, (2) the mechanisms underlying cryptic splicing in TDP-43 proteinopathies and the role of cryptic RNA and proteins in FTLD, (3) the role of the endo-lysosomal system in the development of TDP-43 pathology and neurodegeneration, and (4) the emerging role of TMEM106B fibrillogenesis in diverse neurodegenerative diseases including TDP-43 proteinopathies and tauopathies. We will use a combination of mouse and induced pluripotent stem-cell modeling, transcriptomics, proteomics, histology, and human tissue analyses to carry-out our proposed studies and address new and potentially transformative ideas as they emerge.", "keywords": [ "Acceleration", "Address", "Adopted", "Affect", "Amyotrophic Lateral Sclerosis", "Area", "Behavior", "Biological Markers", "Brain", "C9ORF72", "COVID-19", "Cells", "Clinic", "Clinical", "Collaborations", "Communities", "Dedications", "Deposition", "Development", "Disease", "Disease Progression", "Environment", "Fostering", "Frontotemporal Dementia", "Frontotemporal Lobar Degenerations", "Functional disorder", "Funding", "Funding Opportunities", "Genetic", "Goals", "Heterogeneity", "Histology", "Language", "Mediating", "Molecular", "Monitor", "Motor Neuron Disease", "Mus", "Mutation", "Nature", "Nerve Degeneration", "Neurodegenerative Disorders", "Neurosciences", "PGRN gene", "Pathogenesis", "Pathogenicity", "Pathologic", "Pathology", "Patients", "Personality", "Physicians", "Process", "Productivity", "Proteins", "Proteomics", "Publications", "RNA", "RNA Splicing", "Research", "Resources", "Role", "Science", "Scientist", "Seminal", "Spinocerebellar Ataxias", "System", "Tauopathies", "Techniques", "Translational Research", "Work", "fibrillogenesis", "flexibility", "forging", "human tissue", "improved", "induced pluripotent stem cell", "innovation", "insight", "interest", "mouse model", "neuropathology", "novel", "patient prognosis", "prevent", "programs", "protein TDP-43", "stem cell model", "tau Proteins", "tool", "transcriptomics" ], "approved": true } } ], "meta": { "pagination": { "page": 1405, "pages": 1405, "count": 14046 } } }