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9873 documents

INPP5E Signaling and Treatment in Rheumatoid Arthritis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Shuying Yang · UNIVERSITY OF PENNSYLVANIA · 2024 · $553,248 — Abstract. Rheumatoid arthritis (RA) is a debilitating chronic joint disease, causing functional disability, severe pain, joint deterioration, and substantial socioeconomic burdens. Presently, there is no cure for RA. While some patients manage RA with methotrexate and other disease-modifying anti-rh…

Targeted Delivery of Cas9 to Lung Epithelial Cells using Enveloped Delivery Vehicles

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

JENNIFER A DOUDNA · J. DAVID GLADSTONE INSTITUTES · 2024 · $236,250 — PROJECT SUMMARY/ABSTRACT The rapid development of genome editing tools has outpaced the ability to deliver these molecules specifically to diseased cells in human patients. A major bottleneck in expanding in vivo applications of genome editing is the lack of targeted, cell-specific delivery vehicles…

Novel Targeted Therapeutics for Breast Cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Bulent Ozpolat · METHODIST HOSPITAL RESEARCH INSTITUTE · 2024 · $351,075 — Project Summary Triple negative breast cancer (TNBC) has the worst prognosis and survival in all breast cancer subtypes with currently available standard therapies, representing an unmet therapeutic challenge. Significant heterogeneity (with six genetically defined subgroups) and a lack of FDA-appro…

HIV-1DetectionandEliminationFrom CNS Mononuclear Phagocytes

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Howard E Gendelman · UNIVERSITY OF NEBRASKA MEDICAL CENTER · 2024 · $690,750 — ABSTRACT Mononuclear phagocytes (MP; monocytes, macrophages, dendritic cells. and microglia) serve as human immunodeficiency virus type one (HIV-1) reservoirs, sites of viral persistence and latency, and inducers of end- organ disease. All are commonly linked to HIV-1 pathobiology. However, the key …

Analyzing the role of cAMP and STAT3 signaling in cartilage homeostasis and osteoarthritis development

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Zhaoyang Liu · UNIVERSITY OF SOUTHERN CALIFORNIA · 2024 · $236,917 — Project Summary Osteoarthritis (OA) is the most common form of degenerative joint disease affecting millions of people worldwide and lead to a tremendous financial burden. Currently there are no disease modifying therapies available for OA, due to limited understanding of the genetic factors and pat…

Novel role of Foxc2 in regulating metabolic disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Kui Cui · BOSTON CHILDREN'S HOSPITAL · 2024 · $125,658 — Abstract The long-term goal of this proposal is developed to uncover the molecular mechanisms and identify critical regulators governing lymphatic vascular function in health and disease in hopes of offering potential new therapeutic targets to combat cardiovascular and metabolic disorders. At the t…

New mechanisms by which complementýregulates the pathogenesis of experimental autoimmune uveitis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

FENG C LIN · CLEVELAND CLINIC LERNER COM-CWRU · 2024 · $402,500 — Abstract Autoimmune uveitis, a common cause of blindness, has an unknown etiology and no known cure. We have been studying experimental autoimmune uveitis (EAU) induced in mice deficient in various complement components, inhibitors, or receptors following active immunization with a retinal antigen …

Lipid mediated oral tolerance

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

SATHY VENKAT BALU-IYER · STATE UNIVERSITY OF NEW YORK AT BUFFALO · 2024 · $465,506 — Project Summary: Autoimmune conditions, allergies and immunogenicity against therapeutic proteins are caused by unwanted immune responses against proteins. Our long-term goal is to rationally develop an immunotherapy platform using protein delivery strategies to treat these clinical conditions. We o…

Enhanced APOE2 Expression into Brain for Therapeutic Strategy for Alzheimer's Disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Takahisa Kanekiyo · NORTH DAKOTA STATE UNIVERSITY · 2024 · $525,712 — SUMMARY/ABSTRACT: Alzheimer’s disease (AD) is a progressive neurodegenerative disease that has emerged as the most prevalent form of late-life dementia in humans. The accumulation, aggregation, and deposition of amyloid-β (Aβ) in the brain are central events in AD pathogenesis. Despite intense effor…

The role of signaling adaptor protein epsin in atherosclerosis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Hong Chen · BOSTON CHILDREN'S HOSPITAL · 2024 · $770,203 — PROJECT SUMMARY/ABSTRACT Atherosclerosis is the leading cause of life-threatening coronary heart disease, ischemic stroke, and peripheral arterial disease in the United States. Notably, dyslipidemia remains a major risk factor despite effective lipid- lowering therapies and prevention programs. This…

Cell-Selective Therapies for Coronary Artery Disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Hana Totary-Jain · UNIVERSITY OF SOUTH FLORIDA · 2024 · $476,323 — Atherosclerotic cardiovascular disease (CVD) represents a serious affliction affecting millions globally. Despite recent advances in pharmacological and percutaneous interventions, CVD remains the leading cause of death and disability in the world. One of the main therapeutic challenges facing ather…

Unidirectional delivery of heat to the eyelid by GoldInLens

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

ANUJ CHAUHAN · COLORADO SCHOOL OF MINES · 2024 · $242,946 — Meibomian gland dysfunction (MGD) is the leading cause of the dry eye disease which affects 6 to 7 million people in the United States. The Meibomian glands in MGD dry eye patients are unable to secrete oily lipids resulting in loss of the lipid layer on the surface of tears, leading to an increase …

Intracellular RNA Nanoparticle Therapeutics to Treat Retinal Neovascularization

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

William R. Freeman · UNIVERSITY OF CALIFORNIA, SAN DIEGO · 2024 · $413,374 — ABSTRACT Diabetic retinopathy is a leading cause of blindness in the industrialized world and has a global prevalence of an estimated 95 million people (1). Proliferative diabetic retinopathy (PDR) and diabetic macular edema (DME) originate from persistently elevated glucose levels leading to microv…

CHOLESTEROL METABOLISM IN THE PHARMACOLOGY OF LIPOSOMAL THERAPEUTICS

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Ninh La-Beck · TEXAS TECH UNIVERSITY HEALTH SCIS CENTER · 2024 · $463,889 — ABSTRACT Drug delivery using liposomes increases tumor drug accumulation while sparing normal tissue. Several liposomal chemotherapies are approved to treat cancer. Unfortunately, lipid nanoparticles such as liposomes interact with the immune system and their impact on the tumor immunologic milieu i…

Role of RBP in programming and reprogramming of cardiac fibroblasts

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Jiandong Liu · UNIV OF NORTH CAROLINA CHAPEL HILL · 2024 · $662,922 — Cardiac fibroblasts (CFs) are the major cardiac cell type responsible for producing extracellular matrix (ECM) proteins, forming a structural scaffold crucial for supporting cardiac tissue during development and homeostasis. CFs are also known for their high plasticity, enabling them to swiftly resp…

Cholesterol Removal as a Treatment for Dry Age-Related Macular Degeneration

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Nehal N Mehta · MOBIUS SCIENTIFIC INC. · 2024 · $276,758 — Abstract The leading cause of blindness in people over 50 in industrialized nations including the United States is age-related macular degeneration (AMD). Early AMD is characterized by cholesterol accumulation under the neurosensory retina called sub-retinal drusenoid deposits or under the retinal …

A Bio-orthogonal Targeting System for Precision Drug Delivery to Vascular Endothelium in Transplanted Organs

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Aria Pearlman Morales · BOSTON UNIVERSITY (CHARLES RIVER CAMPUS) · 2024 · $53,974 — Project Summary and Abstract. The viability of a transplanted organ and the patient’s health are inherently limited by the burdens of immunosuppressive therapy, which must be used for the duration of the patient’s life. Systemic administration of these immunosuppressive agents increases the incidenc…

Development of peptoid-based barcodes for in vivo high-throughput screening of targeted nucleic acid delivery

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Jayoung Kim · UNIVERSITY OF NORTH TEXAS HLTH SCI CTR · 2024 · $222,000 — PROJECT SUMMARY / ABSTRACT The proposed project aims to deliver a research tool that supports accurate and resource- efficient biodistribution studies involving a library of gene-encapsulated nanoparticles. The state-of-the-art technology to perform in vivo high-throughput screening of non-viral gen…

Rapid Dehydration and Stabilization of Biopharmaceutical Formulations at Room Temperature

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

Maksim Mezhericher · INAEDIS, INC · 2024 · $312,634 — Abstract INAEDIS proposes to develop a platform technology for rapid room temperature dehydration of vaccines and biopharmaceutical formulations using ultra-fine droplet aerosols, eliminating the need for logistically challenging cold chain infrastructure. Some 20% of pharmaceuticals ‒ around $35B i…

In vivo Sickle Cell Disease Gene Therapy Using a Novel Virus Like Particle

NIH Pandemic-Era Grants · Pandemic Era Grants / 2024

David Scott Johnson · GIGAMUNE, INC. · 2024 · $300,000 — PROJECT SUMMARY Project Title: In vivo Sickle Cell Disease Gene Therapy Using a Novel Virus Like Particle Organization: GigaMune, Inc. PI: David S. Johnson, Ph.D. β-hemoglobinopathies are the most common monogenic disorders worldwide. These autosomal recessive disorders affect the normal production …