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9873 documents

A nanoengineering platform for programmable gene editing therapies against rare diseases

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Steven L Armentrout · PARABON NANOLABS, INC. · 2023 · $325,000 — PROJECT SUMMARY More than 300 million people worldwide are affected by a genetic health condition. Over 4,400 genetic diseases have been identified; nearly all of which are considered rare, which limits the amount of research each receives. Gene therapy is an attractive approach for treatment of gen…

Placental Serum Amyloid A as a Therapeutic Target to Prevent Preterm Birth and Prematurity Related Morbidity

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

IRINA BURD · UNIVERSITY OF MARYLAND BALTIMORE · 2023 · $424,875 — Summary Preterm birth (PTB) and associated fetal brain injury bring about adverse perinatal outcomes including mortality in offspring, and high cost of care and treatment for the family and society. The pathologic immune responses in the placenta during maternal inflammation (MI) are thought to be t…

Computationally-Inspired Design of Non-Viral Gene Delivery Vehicles for mRNA-Based Cystic Fibrosis Therapies

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Shashi Murthy · NANITE INC. · 2023 · $299,995 — PROJECT SUMMARY Cystic fibrosis (CF) is a debilitating and life-shortening disease affecting more than 70,000 people worldwide, with ~1,000 new cases expected to be diagnosed every year. This disease is an autosomal recessive genetic disorder associated with mutations in the CF transmembrane conduct…

BLR&D Research Career Scientist Award Application

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

AJAY NMN RANA · JESSE BROWN VA MEDICAL CENTER · 2023 — Project Summary/Abstract: The main focus of research in our group is to understand the underlying molecular mechanisms of pancreatic and breast cancer pathogenesis. The overarching goal of our research is to investigate how the cellular pathways (i.e. circuits) are dysregulated and can lead to uncon…

Targeting Molecular Transducers of Exercise for Osteoarthritis Therapies

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

TIMOTHY M GRIFFIN · OKLAHOMA CITY VA MEDICAL CENTER · 2023 — Osteoarthritis (OA) disproportionately affects veterans, resulting in more pain and functional limitations compared to the general population. No disease-modifying treatments exist for OA, and current pain medications (e.g., opioids and NSAIDs) have limited long-term efficacy and adverse side effect…

Mechanism of Intratumoral Transport of Particulate Drugs

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Shu-Hsia Chen · METHODIST HOSPITAL RESEARCH INSTITUTE · 2023 · $456,480 — The tumor vasculature is generally considered as leaky, and thus allows accumulation of big molecules and particles within a certain size range to penetrate and retain. Consequently, many cancer drugs have been packaged into simple nanoparticles or composite drug particles in order to improve accumu…

A critical role for macrophage ferroptosis in promoting fungal invasion in lung transplant recipients

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Joe L Hsu · STANFORD UNIVERSITY · 2023 · $609,585 — PROJECT SUMMARY/ ABSTRACT This application focuses on the contribution of transplant hemorrhage-induced iron overload in the dysregulation of pulmonary macrophages (mɸs) and the promotion of invasive aspergillosis. Aspergillus fumigatus (Af) is a ubiquitous mold that releases airborne spores (conidi…

Innate Immunity, Cholesterol, and NASH Pathogenesis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Lucy Mary Golden · UNIVERSITY OF SOUTHERN CALIFORNIA · 2023 · $371,250 — PROJECT SUMMARY /ABSTRACT Non-alcoholic fatty liver disease (NAFLD) is the most common cause of chronic liver disease, affecting at least a quarter of the world’s population, and therefore highly relevant to the NIH mission. From steatosis to fibrosis, hepatic inflammation drives disease progression…

Nanoparticle-based host-directed therapies for eradication of Mycobacterium tuberculosis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Admire Dube · STELLENBOSCH UNIVERSITY · 2023 · $318,225 — SUMMARY Tuberculosis remains a major global public health threat. Although relatively effective drug regimens are available, treatment failure remains a major roadblock to tuberculosis control. This is in part due to a high incidence of drug-resistant Mycobacterium tuberculosis (Mtb) strains, as wel…

Nanotechnology-based platform for the development of next-generation vaccines against opioid use disorder (OUD)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Fatima Alamin Awad Alkareem Hamid · UNIVERSITY OF MINNESOTA · 2023 · $34,953 — Project Abstract: The highly complex OUD and overdose epidemic poses a huge public health and economic burden. Current FDA-approved pharmacotherapies against OUD and overdose use opioid receptor agonists and antagonists. These therapies show overall limited efficacy, due to their side effects, subop…

Sustained release of fenofibrate for the treatment of diabetic retinopathy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jian-Xing Jay Ma · VIRGINIA COMMONWEALTH UNIVERSITY · 2023 · $538,028 — PROJECT SUMMARY Diabetic retinopathy (DR) is the most common cause of severe vision loss in the working-age population in the world. However, clinical studies have shown that approximately 40% DR patients do not adequately respond to anti-VEGF drugs. There is no drug treatment for those 40% DR pati…

Establishing a novel gene editing strategy for BBS7 using human retinal organoids

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Kathleen R Chirco · OREGON HEALTH & SCIENCE UNIVERSITY · 2023 · $249,000 — As the leading cause of inherited retinal degeneration, retinitis pigmentosa (RP) affects about 1.5 million people worldwide. Bardet-Biedl syndrome (BBS) is the second most common causes of syndromic RP, and is characterized as an autosomal recessive ciliopathy with severe photoreceptor degeneration…

Mast Cell Activation as a Common Mechanism of Pulmonary Toxicity by Chemical Threat Agents

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

ALISON K BAUER · UNIVERSITY OF COLORADO DENVER · 2023 · $462,501 — Project Abstract The Department of Homeland Security considers numerous chemical threat agents a concern for human health, specifically those that are acute pulmonary toxicants. In acute lung injury, inflammation is critical thus we propose that inflammation is a common mechanism of lung injury caus…

Novel Formulation Technology to Enhance Oral Absorption of Water-insoluble Drugs

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Xiaowei Dong · UNIVERSITY OF NORTH TEXAS HLTH SCI CTR · 2023 · $66,958 — Project Summary/Abstract Bioavailability enhancement technologies for insoluble drugs represent incremental progress, but no breakthrough oral drug technologies have been identified in the past three decades. Lipid-based drug delivery systems (LBDDS) are considered as a well-established, very import…

miRNA-Nanotechnology as a novel regenerative therapy for lymphangioleiomyomatosis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jacob Brenner · BIOSPUTNIK LLC · 2023 · $301,016 — ABSTRACT Lymphangioleiomyomatosis (LAM) is a Tuberous sclerosis-related disorder. Both occur due to an inherited or sporadic mutation in either the TSC1 or TSC2 gene, which function as negative regulators of the mTOR pathway. Uncontrolled mTORC1 activity leads to the neoplastic proliferation of abno…

Gene Therapy for Inherited Blood Disorders

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Andre LaRochelle · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · 2023 · $1,332,597 — Objective 1: Develop novel HSPC gene correction strategies Because multiple causative mutations have been identified in FA, targeted introduction of a therapeutic open reading frame at the endogenous FANCA genetic locus is desirable to allow single-construct genetic correction regardless of th…

Phage-inspired engineering and evolution

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Irene Ann Chen · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2023 · $383,143 — Project Summary Phages are viruses that infect bacteria, and are the most abundant type of organism on earth. In addition, phages are a potential antibacterial therapy. The overall focus of the project is phage- inspired engineering for antibacterial applications and fundamental evolutionary studies…

Nanodisc-displayed Protein Vaccines

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Aleksandra Elzbieta Sikora · HENRY M. JACKSON FDN FOR THE ADV MIL/MED · 2023 · $197,239 — ABSTRACT Neisseria gonorrhoeae (Ng) is a human-specific pathogen and the etiological agent of gonorrhea, a sexually transmitted infection with a significant global health burden of ~78 million new cases annually. While often asymptomatic, untreated gonorrhea can lead to pelvic inflammatory disease, …

IN UTERO SMALL AND LARGE ANIMAL RESOURCE CORE

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Alan W. Flake · CHILDREN'S HOSP OF PHILADELPHIA · 2023 · $1,069,284 — PROJECT SUMMARY Many monogenic diseases strike prior to or soon after birth and cause significant morbidity and mortality. In utero genome editing (IUGE) takes advantage of normal fetal properties, including small size, abundant progenitor cells, and immature immune system, to safely and efficiently…

Clinical Trials Program

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

TIMOTHY J. WILKIN · WEILL MEDICAL COLL OF CORNELL UNIV · 2023 · $954,399 — The overall goal of the proposal is to advance prevention interventions for HPV-associated cancers occurring among men and women living with HIV in Latin America and the Caribbean (LAC). We are focusing on prevention of two HPV-associated cancers, oropharyngeal cancer and cervical cancer. Both cance…