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9873 documents

Nanoformulated CRISPR Ribonucleoproteins for Ultrasound-Facilitated Brain Gene Editing

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Yeh-Hsing Lao · STATE UNIVERSITY OF NEW YORK AT BUFFALO · 2023 · $442,000 — Nanoformulated CRISPR Ribonucleoproteins for Ultrasound-facilitated Brain Gene Editing Abstract Emerging CRISPR technologies provide new opportunities to advance gene therapy in treating many intractable genetic diseases, including neuronal degeneration disorders. Given the failures of clinical tri…

Development of phospholipid-based nanotherapeutics for treating abdominal aortic aneurysm

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Yanhong Guo · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2023 · $763,935 — PROJECT SUMMARY/ABSTRACT Abdominal aortic aneurysm (AAA) is defined as a permanent, localized dilatation of the abdominal aorta with the potentially fatal consequence of aortic rupture. The only effective treatment for AAA is open or endovascular surgical repair when AAA patients with symptomatic or…

Nanodisc Platform for 19F-MRI

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Nicholas Oliver Fischer · UNIVERSITY OF CALIFORNIA AT DAVIS · 2023 · $272,874 — 19F MRI, has shown promising success in clinical trials for monitoring cell therapy, where small groups of cells, showing poor contrast in 1H-MRI (Magnetic Resonance Imaging), are easily tracked using 19F-MRI.1 Contrast is provided by nanoemulsions of perfluorocarbon (PFC) oils pre-loaded into cells…

A novel bioengineering approach to restoring permanent periodontal inflammatory bone loss

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Hae Lin Jang · BRIGHAM AND WOMEN'S HOSPITAL · 2023 · $834,068 — About 80% of Americans experience periodontitis in their lifetime. Alveolar bone loss leads to loosening or loss of teeth or dental implants that disrupts the most basic daily functions, such as eating and speaking. Various bone grafts are being used to restore alveolar bone loss, but poor prognosis…

Hepatic lymphatics in alcohol-associated liver disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

YASUKO IWAKIRI · YALE UNIVERSITY · 2023 · $211,584 — SUMMARY A subset of patients with alcohol-associated liver disease (ALD) develop severe alcoholic hepatitis (AH), carrying a very poor prognosis. Control of inflammation is a key to the treatment of AH. Hepatic lymphatics are highly involved in the regulation of the inflammatory response by dischar…

Understanding and Controlling the Contribution of Fibrinolysis to Bleeding Using a Long-Acting Antifibrinolytic RNA Therapy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Christian Kastrup · VERSITI BLOOD HEALTH, INC. · 2023 · $757,275 — SUMMARY BACKGROUND: The balance between clot formation (coagulation) and degradation (fibrinolysis) is disrupted in patients with bleeding disorders or severe hemorrhage, resulting in increased bleeding. The extent to which fibrinolysis contributes to bleeding from the onset of acute bleeding in the…

Project 1. Design of immunogens that bind to the UCA and to IAs and mature DH511 bnAbs in optimal affinities

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

S. Munir ALAM · DUKE UNIVERSITY · 2023 · $325,000 — Induction of broadly neutralizing antibodies (bnAbs) is a critical unmet goal of HIV vaccine development. BnAb DH511 is of high interest as a vaccine lead due to its very high breadth and the high in vivo protective potency of MPER bNAbs. Key challenges for inducing DH511-like bnAbs are: (a) the low…

Molecular Genetics Of Heritable Human Disorders

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

JANICE CHOU · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · 2023 · $1,942,202 — Clinically, GSD-Ib patients manifest a metabolic phenotype of impaired blood glucose homeostasis and a long-term risk of hepatocellular adenoma/carcinoma (HCA/HCC). The etiology of HCA/HCC in GSD-Ib is unknown. Studies have shown that deficiency in autophagy, an evolutionary conserved, degradative p…

Omics Core

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Xiang Zhang · UNIVERSITY OF LOUISVILLE · 2023 · $380,400 — The primary function of OMICS Core is to provide members of the University of Louisville Alcohol Research Center (ULARC) with the expertise, resources, and facilities needed for metabolomic, proteomic, and bioinformatic analyses of biological samples collected to study nutrition, gut flora/intestina…

IND-enabling development of Radioprotectin 1: a dual GI/HE radiation mitigator

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

GABOR J TIGYI · UNIVERSITY OF MARYLAND BALTIMORE · 2023 · $310,386 — The US population at large, and particularly military personnel and first responders, are at risk of radiation expo-sure due to the explosion of a nuclear device, a nuclear reactor accident, and the threat of radiation terrorism. There is no radiation medical countermeasure (RCM) drug approved by t…

Develop an engineered Cas effector for in vivo cell-targeted delivery in the eye to treat autosomal dominant BEST disease

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

David M Gamm · UNIVERSITY OF WISCONSIN-MADISON · 2023 · $1,360,328 — PROJECT SUMMARY/ABSTRACT – FOLLOWER PROJECT 2 Best Disease (BD) is a prevalent, primarily autosomal dominant, macular degenerative disorder that results in retinal pigment epithelium (RPE) dysfunction and progressive loss of central vision. BD results from >200 known missense mutations in the BEST1 …

Improving response durability after CAR T cell therapy for multiple myeloma

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Michael C. Milone · UNIVERSITY OF PENNSYLVANIA · 2023 · $506,376 — PROJECT SUMMARY / ABSTRACT (PROJECT 3) Multiple myeloma (MM) is a hematologic malignancy of bone marrow plasma cells. Even though MM is almost universally fatal, modern therapy has markedly improved survival. Among the most promising new MM therapies are CAR T cells targeting B cell maturation antig…

Project 3: Skin hypoxia, MCPyV infection, and MCC tumorigenesis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jianxin You · UNIVERSITY OF PENNSYLVANIA · 2023 · $418,564 — Project Abstract Merkel cell carcinoma (MCC) is one of the most aggressive skin cancers. Clonal integration of Merkel cell polyomavirus (MCPyV) genome into the host DNA has been observed in ~80% of MCCs, and represents a key causal factor for MCC development. LT and sT encoded by MCPyV genome have b…

Nanoparticle-based development for HIV-1 biomolecule delivery

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

THOMAS HASSELL · INTERNATIONAL AIDS VACCINE INITIATIVE · 2023 · $635,012 — NIAID Process Development and Analytical Support for Development of HIV Vaccines: The development of vaccines to prevent the spread of HIV infection is among the National Institute of Allergy and Infectious Diseases (NIAID) highest priorities. While advances in immunology and molecular biology conti…

Investigation of the role of VEGFA in harnessing cholangiocyte-driven liver regeneration

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

VALERIE B GOUON-EVANS · BOSTON MEDICAL CENTER · 2023 · $577,631 — PROJECT SUMMARY / ABSTRACT Liver disease affects hundreds of millions of people worldwide. Liver transplantation remains the only treatment for end stage liver disease (ESLD); however, transplantation faces a major burden due to a shortage of liver donors. The liver is known for a remarkable regener…

Long-Acting RNAi Therapy for Atherosclerosis and Insulin Resistance

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Jinjun Shi · BRIGHAM AND WOMEN'S HOSPITAL · 2023 · $682,727 — ABSTRACT With the ability to silence individual genes and to drug the ‘undruggable’, RNA interference (RNAi) therapy has recently shown clinical success by delivering small interfering RNA (siRNA) to the liver for genetic diseases. However, new delivery strategies will be needed to expand the target…

Biomimetic Macrophage Membrane-Coated Nanosponges: A Novel Therapeutic for Multidrug-Resistant Pseudomonas aeruginosa and Acinetobacter baumannii Hospital-Associated Pneumonia

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

Angela Meier · UNIVERSITY OF CALIFORNIA, SAN DIEGO · 2023 · $1,027,000 — PROJECT SUMMARY Pneumonia is the most common cause of hospitalization due to infection in the US and the most common cause of infection-related death. Mortality in healthcare-associated pneumonia (HAP) is 13% overall and 36% in patients admitted to the ICU. Bacterial lung infections are also a frequ…

BLR&D Research Career Scientist Award Application

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

SEBASTIAN JOYCE · VETERANS HEALTH ADMINISTRATION · 2023 — The  overarching  goal  of  my  research  program  is  to  understand  the  molecular  mechanisms  of  immune  surveillance mediated by tissue-­resident memory T cells that patrol the mucosal barrier of the lungs to control  infectious diseases and cancers. My laboratory is best recognized for its c…

Cell targeting with synthetic sense-and-respond protease circuits

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

MICHAEL B ELOWITZ · CALIFORNIA INSTITUTE OF TECHNOLOGY · 2023 · $549,984 — Cell targeting with synthetic sense-and-respond protease circuits Abstract A fundamental challenge in biomedicine is developing therapeutics that can target specific cell populations. Synthetic protein circuits, based on engineered proteins that interact with one another and with endogenous cellular…

Novel Nanoparticle Respiratory Tract Mucosal Vaccine

NIH Pandemic-Era Grants · Pandemic Era Grants / 2023

FREDERICK D QUINN · UNIVERSITY OF GEORGIA · 2023 · $479,566 — Summary Bacterial and viral respiratory pathogens interact closely with mucosal surfaces. Specialized innate and adaptive mucosal immune systems protect these surfaces and are the first line of defense for the body. One of the more important reasons for the development of mucosal vaccines is the inc…