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9873 documents

Targeting MED31-driven transcription recycling in lethal prostate cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Qianben Wang · DUKE UNIVERSITY · 2023 · $507,799 — Project Summary/Abstract Patients with lethal castration-resistant prostate cancer (CRPC) are currently treated with agents targeting androgen receptor (AR) signaling. However, AR inhibition has not dramatically improved CRPC patient survival, underscoring the need to discover novel oncogenic mechan…

Nanoparticle-mediated reduction of oxidative stress for the treatment of traumatic brain injury

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Forrest M Kievit · UNIVERSITY OF NEBRASKA LINCOLN · 2023 · $439,444 — PROJECT SUMMARY/ABSTRACT Traumatic brain injury (TBI) is the leading cause of disability and death in people under 45 with approximately 10 million new cases each year worldwide. The effects of TBI can be severe, including neurocognitive, physical, and psychosocial impairment. There remains a signi…

Placental identified NHIP regulating neuronal oxidative stress in autism

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Janine M LaSalle · UNIVERSITY OF CALIFORNIA AT DAVIS · 2023 · $666,137 — The human fetal brain consumes up to 60% of the body’s oxygen and energy consumption, despite making up ~13% of body mass. When the demand for oxygen in the placenta and developing brain exceeds its supply, hypoxia is induced, followed by changes to mitochondrial respiration, protein translation, an…

Nonviral delivery of CRISPR-Cas9 into hepatocytes combined with APAP selection for treatment of Familial Hypercholesterolemia

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Renee Nicole Cottle · CLEMSON UNIVERSITY · 2023 · $565,787 — PROJECT SUMMARY Familial hypercholesterolemia (FH) affects 1 in 250 people and is characterized by impaired low-density lipoprotein (LDL) metabolism resulting in premature cardiovascular disease. CRISPR-Cas9-induced loss of function mutations in the gene encoding Angiopoietin-like 3 (ANGPTL3) has be…

Developing and Automating an Extracellular Vesicle-Based Test for Early Detection of Hepatocellular Carcinoma

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Han-Yu Chuang · EXIMIUS DIAGNOSTICS CORP · 2023 · $661,609 — PROJECT SUMMARY Hepatocellular carcinoma (HCC) comprises 80-85% of primary liver cancers and frequently develops in patients with liver cirrhosis or chronic hepatitis B virus infection. HCC's poor prognosis is primarily due to advanced-stage diagnosis. Current clinical practice guidelines recommend …

Generation of Hematopoietic Stem and Progenitor Cells from Human iPSCs

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Andre LaRochelle · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · 2023 · $1,332,597 — Objective 1: Develop a scalable culture system for hematopoietic differentiation of hiPSCs To address the limitations of current iPSC differentiation approaches, we initially sought to develop a chemically-defined and scalable hematopoietic differentiation protocol requiring no replating, EB format…

Click Chemistry-Mediated Surface Protein Assay for Quantifying Subpopulations of Hepatocellular Carcinoma-associated Extracellular Vesicles

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Vatche Agopian · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2023 · $674,097 — PROJECT SUMMARY Hepatocellular carcinoma (HCC) accounts for 80-85% of primary liver cancers, and mainly occurs in patients with liver cirrhosis or chronic hepatitis B virus (HBV) infection. Prognosis of HCC is dismal primarily due to advanced stage of disease at diagnosis. Current clinical practice …

Development of a first-in-class antiviral to address CMV drug resistance in immunocompromised patients

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Robert Rodick · VXBIOSCIENCES, INC. · 2023 · $300,000 — ABSTRACT Human cytomegalovirus (CMV) infects a majority of the world’s population and is a leading cause of disease in transplant patients and newborns, accounting for more congenital birth defects than Down’s syndrome, spina bifida, or fetal alcohol syndrome. There is no approved vaccine and all cu…

In vivo precision genome editing to correct genetic disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Gregory A. Newby · JOHNS HOPKINS UNIVERSITY · 2023 · $249,000 — PROJECT SUMMARY Genetic diseases impact over 1 in 50 newborns worldwide and yet there are no approved therapies capable of correcting the underlying genetic defects. As a result, most patients continue to suffer throughout their life and require frequent interventions to ameliorate symptoms. I aim …

Novel formulation technology for the sustained release naloxone to improve outcomes in the management of opioid overdose

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Cesar Torres Luna · LYNTHERA CORPORATION · 2023 · $320,000 — PROJECT SUMMARY Opioid overdose was responsible for less than 10,000 deaths in 1999 but increased to nearly 50,000 by 2019. Data reported by the Center for Diseases Control and Prevention's National Center for Health Statistics showed that the 12-month period leading up to April 2021 had more than 1…

The regulation and functions of Group 1 CD1-restricted T cells

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Chyung-Ru Wang · NORTHWESTERN UNIVERSITY AT CHICAGO · 2023 · $665,738 — PROJECT SUMMARY Group 1 CD1-restricted T cells are members of the unconventional T cell family that recognize self- and microbial lipid antigens presented by CD1a, CD1b, and CD1c molecules. Group 1 CD1-restricted T cells have been implicated to play critical roles in various autoimmune and infectiou…

Therapeutic targets and novel anticancer agents for endocrine cancers

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Naris Nilubol · DIVISION OF BASIC SCIENCES - NCI · 2023 · $891,488 — 1. Identification of novel synergistic drug combination therapy for ACC 1.1 Status: Published [J Exp Clin Cancer Res 2022]. The synergy of MELK and CDK inhibitors targeting multiple clinically-relevant molecules in ACC. Clinical trial development is ongoing. 1.2 The novel synergistic combination of …

LEAD PROJECT 1: PHENYLKETONURIA (PKU)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Kiran Musunuru · CHILDREN'S HOSP OF PHILADELPHIA · 2023 · $1,069,284 — 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 …

PROJECT 2: HEREDITARY TYROSINEMIA TYPE 1 (HT1)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

William H. Peranteau · CHILDREN'S HOSP OF PHILADELPHIA · 2023 · $1,069,283 — 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…

Combinatorial targeting for the treatment of B-cell acute lymphoblastic leukemia

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Sharan Kumar Reddy Bobbala · WEST VIRGINIA UNIVERSITY · 2023 · $235,717 — B-cell acute IympnooIast1c IeuKemIa (ALL) accounts for 75% of all ALL diagnoses. Specifically, treating the Philadelphia {Ph) chromosome subgroup (which creates a new gene called BCR-ABL) of B-cell ALL challenging and has high relapse rates and poor survival. Mounting evidence from recent findings s…

Research Project 3: siRNA Therapy and Small Molecule Combination Treatment for Filoviruses

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Thomas William Geisbert · UNIVERSITY OF TEXAS MED BR GALVESTON · 2023 · $723,636 — PROJECT SUMMARY - Research Project 3 Members of the filovirus genera Ebolavirus and Marburgvirus are HHS Tier 1 Category A priority pathogens that cause severe and often fatal hemorrhagic fever (HF) in humans and nonhuman primates (NHPs). There are presently no approved therapeutics for ebolavirus…

Targeted Nano-drug-delivery-systems of Frist-in-class Drugs for CRPC: PK/PD Evaluation and Combination Therapy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Huan Xie · TEXAS SOUTHERN UNIVERSITY · 2023 · $170,518 — PROJECT SUMMARY Prostate cancer (PCa) is the second leading cause of cancer death in the United States among men and it is more common in African American males. Despite initial good responses to androgen deprivation or anti- androgen drugs, tumors invariably recur and develop into lethal castration…

Lipid Based Self Assembled Materials Synthesis and Characterization

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Colby Shad Thaxton · NORTHWESTERN UNIVERSITY AT CHICAGO · 2023 · $275,984 — SUMMARY The overall goal of the Lipid-Based Self-Assembled Materials and Characterization Core (LBSAMCC) is to synthesize, characterize, and deliver highly novel therapeutics for ameliorating the devastating effects of nitrogen and sulfur mustards to the eye (Project 1) and skin (Project 2). High-de…

Adjuvant Comparison and Characterization (HIV)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

BALI PULENDRAN · STANFORD UNIVERSITY · 2023 · $1,780,989 — The goal of the Adjuvant Comparison and Characterization (ACC) program is to support side-by-side comparison of adjuvants in combination with clinically relevant vaccine/antigen platforms, and to establish both systemic and tissue-specific immunological profiles (“immune fingerprints”) of adjuvants …