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9873 documents

PROJECT 2: HEREDITARY TYROSINEMIA TYPE 1 (HT1)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

William H. Peranteau · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $1,630,863 — PROJECT SUMMARY Hereditary tyrosinemia type 1 (HT1) is an autosomal recessive metabolic liver disease that can cause death in the first months of life and incurs an increased risk of hepatocellular cancer. The current treatment option—strict adherence to twice daily dosing with NTBC, a repurposed he…

Polymeric Materials Synthesis and Characterization

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Nathan Claude Gianneschi · NORTHWESTERN UNIVERSITY AT CHICAGO · 2024 · $319,998 — Project Summary The Polymeric Materials Synthesis and Characterizations Core will formulate, synthesize, and characterize lead candidates for the mitigation of mustards and provide support and chemical expertise to other cores and projects. The Core will be co-directed by Drs. Nathan Gianneschi (Dep…

Design and Engineering of VesiVax HIV Immunogen Formulations

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Samon Ho · MOLECULAR EXPRESS, INC. · 2024 · $1,999,866 — Particle-based Co-Delivery of HIV Immunogens as Next-Generation HIV Vaccines: A major focus of HIV vaccine research has been the development of immunogens that elicit broadly neutralizing antibody responses targeting the envelope protein (Env). While the field has predominantly focused on immunogen…

BioSAXS

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Richard Edward Gillilan · CORNELL UNIVERSITY · 2024 · $1,589,509 — Project summary: The MacCHESS Synchrotron Source for Structural Biology facilitates the utilization of both established and emerging technologies to enable the structural characterization of proteins involved in critically important biological processes with significant consequences for diseases su…

LEAD PROJECT 1: PHENYLKETONURIA (PKU)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Kiran Musunuru · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $1,419,953 — PROJECT SUMMARY Phenylketonuria (PKU) is an autosomal recessive disorder caused by mutations in the gene encoding phenylalanine hydroxylase (PAH), resulting in the accumulation of phenylalanine (Phe) to neurotoxic levels. Among the five most frequently occurring pathogenic PAH variants worldwide is …

Signal transduction mechanisms that mediate normal and pathologic angiogenesis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jinjiang Pang · UNIVERSITY OF ROCHESTER · 2023 · $471,903 — Title of the grant Signal transduction mechanisms that mediate normal and pathologic angiogenesis Abstract Pulmonary arterial hypertension (PAH) is a progressive disease, characterized by vasoconstriction, cell proliferation, and fibrosis, leading to elevated pulmonary arterial pressure and often ca…

Adipocyte to neuron signaling in thermogenic programming of white adipose tissue

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

MICHAEL P CZECH · UNIV OF MASSACHUSETTS MED SCH WORCESTER · 2023 · $582,900 — Abstract The overarching long term goal of our laboratory is to understand and exploit how adipose tissues exert powerful control over whole body glucose tolerance and insulin sensitivity. Small amounts of mouse brown (BAT) or human “Beige” adipocytes transplanted into recipient mice can improve glu…

Bionanotechnology approach for treatment of lung cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Tamara Minko · RUTGERS BIOMEDICAL AND HEALTH SCIENCES · 2023 · $581,249 — Project Title: Bio-nanotechnology approach for treatment of lung cancer Project Summary Lung cancer is the leading cause of cancer-related deaths worldwide. Non-Small Cell Lung Carcinoma (NSCLC) is the most common type of lung cancer, accounting for more than 80% of all lung cancer cases. Chemothera…

Barrier Damage and The Immune CascadeNorthwestern University CounterACT Center of Excellence (NUCCX)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

ROBERT M LAVKER · NORTHWESTERN UNIVERSITY AT CHICAGO · 2023 · $2,683,799 — Northwestern University (NU) is recognized for its strong interdisciplinary cutaneous, ocular, nanotechnology and immunology research programs with particular strength in investigations related to wound repair and inflammation. Leveraging these strengths, the theme of the NU CounterAct Center of Exc…

Engineered Nanoformulation for Immune-modulation in Cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Rajagopal Ramesh · UNIVERSITY OF OKLAHOMA HLTH SCIENCES CTR · 2023 · $543,107 — Expression of immune check point (ICP) molecules on tumor cells and the host immune cells, especially T-cells present in the tumor milieu, negatively impact the cancer treatment outcome resulting in inefficient tumor eradication. Data also exist showing chemo- and radio- therapy induce ICP proteins …

Gene Therapy Design Principles for Duchenne Muscular Dystrophy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Asuka Eguchi · UNIVERSITY OF CALIFORNIA-IRVINE · 2023 · $162,000 — Project Summary/Abstract The long-term objective of this study is to develop gene therapies that treat Duchenne muscular dystrophy (DMD) cardiomyopathy. DMD cardiomyopathy, characterized by ventricular chamber enlargement and thinning of the ventricular wall, ultimately leads to heart failure. Patho…

Ligand-Directed KRAS G12V Mutant-Specific Therapeutics

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Chad V Pecot · ENFUEGO THERAPEUTICS INC. · 2023 · $275,645 — Project Summary The proto-oncogene KRAS is one of the most critical genes in cancer, yet, as a drug target, it has proven to be among the most elusive. A remarkable 30% of lung adenocarcinomas, 45% of colon, and 98% of pancreatic cancers are driven by KRAS mutations. These cancers account for the t…

Understanding the Relationship LNP Structure, Cholesterol Trafficking, and InVivo Delivery

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

James Dahlman · EMORY UNIVERSITY · 2023 · $398,402 — Project Summary Scientists can create thousands of chemically distinct nanoparticles using a growing number of high throughput chemistries, but it is still difficult to test more than a few nanoparticles in vivo. The goal of this work is to substantially improve how lipid nanoparticles (LNPs) delive…

Continued Development and Maintenance of the UltraScan Software

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Emre H. Brookes · UNIVERSITY OF MONTANA · 2023 · $293,074 — Project Summary/Abstract We propose to continue development and support for the UltraScan-III (US3) software suite and develop new code and methods to address challenging biomedical research questions that can be solved with the latest instruments available for analytical ultracentrifugation and at …

Dual action immunostimulatory nanoparticles for treatment of aggressive cancers

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Efstathios Karathanasis · CASE WESTERN RESERVE UNIVERSITY · 2023 · $600,913 — PROJECT SUMMARY: Cancer immunotherapy is based on the premise of immune-recognition and targeted killing of tumor cells, thus possesses the promising power to eradicate aggressive disease. Notably to date, immunotherapy with immune checkpoint inhibitors significantly prolongs the survival of patient…

The Role of Nutrition in the Development/Progression of Alcohol-Induced Organ Injury

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

CRAIG J. MCCLAIN · UNIVERSITY OF LOUISVILLE · 2023 · $1,386,365 — The University of Louisville Alcohol Research Center (ULARC) was created to serve as a regional/national resource to perform interdisciplinary collaborative research on the role of nutrition as a cofactor in alcohol induced organ injury and as a possible therapeutic intervention to prevent/treat thi…

A Label-free Extracellular-vesicle Automated Purification System (LEAP System)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Yuchao Chen · WELLSIM BIOMEDICAL TECHNOLOGIES, INC. · 2023 · $749,163 — PROJECT SUMMARY / ABSTRACT Extracellular vesicles (EVs) are small lipid bilayer particles secreted by most cell types, which encapsulates biomolecules including lipids, proteins, and nucleic acids from cells of origin. Profiling of EV-derived biomarkers provides a promising approach for diagnosis of…

Systemic RNA Delivery to Tumors

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jinjun Shi · BRIGHAM AND WOMEN'S HOSPITAL · 2023 · $546,127 — ABSTRACT The use of RNA technologies to specifically target genetic alterations in tumor cells has shown great potential of becoming a novel therapy modality for cancer treatment. Nevertheless, systemic delivery of RNA agents such as messenger RNA (mRNA) to tumor cells in vivo commonly faces multipl…

Regulatory Role of Transferrin in Erythropoiesis and Iron Metabolism

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Robert E Fleming · SAINT LOUIS UNIVERSITY · 2023 · $708,121 — ABSTRACT The overall goal of our proposal is to investigate how iron metabolism and erythropoietic activity are interrelated, and the mechanisms whereby dysregulation in one contributes to pathophysiology in the other. Roles for the two transferrin receptors in these processes is clear; however, lit…

Mechanisms of Pro-Resolving Mediators in Periodontal Regeneration

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

THOMAS Elliott VAN DYKE · ADA FORSYTH INSTITUTE, INC. · 2023 · $463,125 — Project Summary Uncontrolled inflammation is a major impediment to tissue engineering, regeneration and reconstruction of both diseased and injured tissues resulting in further tissue injury, tissue scarring and fibrosis. Stem cell activity is disrupted by persistent signals promoting inflammation, …