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9873 documents

Engineered nanoparticles to rescue complement dysfunction and vascular disease during diabetes

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Gary Liu · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · 2022 · $69,802 — PROJECT SUMMARY/ABSTRACT Vascular injury and disease during diabetes lead to end-organ dysfunction (kidney failure, blindness, amputation) and cardiovascular disease that profoundly reduces patient quality of life and life expectancy. The proposed work seeks to develop new therapeutics to prevent an…

Novel hybrid molecule with both IOP lowering and neuroprotective effects for treatment of glaucoma

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Suchismita Acharya · UNIVERSITY OF NORTH TEXAS HLTH SCI CTR · 2022 · $358,900 — Glaucoma is a neurodegenerative disease of the eye with an estimated prevalence of 80 million patients worldwide by 2020, at least 6 to 8 million becoming bilaterally blind. Elevated intraocular pressure (IOP) causing axonal degeneration of the optic nerve and progressive loss of retinal ganglion ce…

Development of STAR Editors (CRISPR-Cas9/lgRNA-ssDNA) for the cure of chronic hepatitis B

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Ju-Tao Guo · GENELANCET BIOSCIENCES INC · 2022 · $300,000 — Abstract This phase 1 SBIR project is to investigate the feasibility of an innovative CRISPR-Cas9 scar-less editing technology to permanently inactivate both hepatitis B virus (HBV) covalently closed circular DNA (cccDNA) and integrated DNA for the cure of chronic hepatitis B (CHB). Although the cur…

PA21259, SBIR Phase I, Developing a RIG-I agonist to prevent COVID-19

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Bryan Berube · HDT BIO CORPORATION · 2022 · $299,984 — Project summary: Despite widespread availability of vaccines, the SARS-CoV-2 pandemic continues to wreak havoc on our healthcare system and our economy. High numbers of unvaccinated individuals combined with vaccine breakthrough cases continues to allow the virus to spread throughout the community a…

Nucleic acid modulators and theranostics for ADAR

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Guizhi Zhu · VIRGINIA COMMONWEALTH UNIVERSITY · 2022 · $113,890 — Project Summary The objectives of this MIRA application are to 1) in vivo profiling of heterogeneous Adenosine deaminase acting on RNA (ADAR) using RNA nano-reporters as a tool to understand ADAR biology and guide the design of ADAR- based therapy; 2) promoting endogenous ADAR modulation using chime…

Ionizable lipid nanoparticles for the delivery of mRNA for CAR T cell engineering

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Margaret M. Billingsley · UNIVERSITY OF PENNSYLVANIA · 2022 · $31,380 — PROJECT SUMMARY CAR T cell immunotherapy is FDA approved for the treatment of acute lymphoblastic leukemia (ALL) and large B cell lymphoma and has shown success in inducing durable remission. However, the therapy is also associated with causing severe, life-threatening side effects—including cytokin…

Polymer-Lipid Particles investigated by Magnetic Resonance Spectroscopy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Dominik Konkolewicz · MIAMI UNIVERSITY OXFORD · 2022 · $427,996 — Project Summary/Abstract Membrane proteins represent approximately 30% of all known proteins but only approximately 1% of all solved protein structures. Despite recent advances in methods for membrane protein structural biology, knowledge about this important class of proteins lags behind their sol…

Smart nanoparticles regulating oncogenic IncRNA for breast cancer therapy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

ZHENG-RONG LU · CASE WESTERN RESERVE UNIVERSITY · 2022 · $350,213 — The goal of this project is to develop smart dual-targeted lipid ECO/siRNA self-assembly nanoparticles to target oncogenic long non-coding RNAs (lncRNAs) as a novel therapy to treat metastatic and drug-resistant triple negative breast cancer (TNBC). Metastasis and drug resistance are the main causes…

"NextGen Long-acting Platform: Targeted Combination Antiretrovirals"

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Rachel Ann Bender Ignacio · UNIVERSITY OF WASHINGTON · 2022 · $1 — The Targeted Long-acting Combination Antiretroviral Therapy (TLC-ART) Program has discovered a novel platform that can stabilize insoluble and soluble antiretroviral drugs together in a nanosuspension which provides long-acting (LA) plasma and cell concentrations in pre-clinical studies. The lead fo…

Construction of in vivo mRNA delivery systems

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Yizhou Dong · OHIO STATE UNIVERSITY · 2022 · $190,245 — Project Summary Cell-specific drug delivery represents one of the most important research areas in the field of drug delivery. Particularly, there are formidable challenges for in vivo mRNA delivery. For example, therapeutic window for current delivery systems is relatively narrow. A large number o…

Nanoparticles based mRNA delivery for treatment of cystic fibrosis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Gaurav Sahay · OREGON STATE UNIVERSITY · 2022 · $658,627 — ABSTRACT: Cystic fibrosis (CF) is conferred by any of ≥1200 known mutations in the gene encoding the CFTR, an ion transporter protein, but in principle, a single gene therapy agent that delivers expression of functional CFTR could treat. Prior attempts to do so have used adeno-associated viral or li…

Protecting the Diabetic Skeletal Muscle by Nampt Activation

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Marco Brotto · UNIVERSITY OF SOUTH FLORIDA · 2022 · $407,887 — Project Summary Diabetes is a leading cause of death in the US and worldwide with deleterious consequences to the musculoskeletal system. Regardless of glycemic control, diabetes in skeletal muscle manifests with altered metabolism leading to progressive skeletal muscle loss, functional decline, fas…

Lipid mediated oral tolerance

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

SATHY VENKAT BALU-IYER · STATE UNIVERSITY OF NEW YORK AT BUFFALO · 2022 · $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…

Nonviral delivery techniques for in vivo prime editing

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

DANIEL G ANDERSON · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · 2022 · $387,750 — Gene editing is a promising strategy for treating or even permanently curing genetic diseases. In particular, a new technique called prime editing has the potential to make small targeted insertions, deletions, and substitutions with very high potential coverage of known disease- causing mutations, …

Defining the molecular interactions within nanoparticles that enable delivery of long nucleic acids

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Daniel John Siegwart · UT SOUTHWESTERN MEDICAL CENTER · 2022 · $419,679 — Project Summary CRISPR/Cas-based gene editing has ushered in a hopeful era that dreams of new therapies for currently untreatable genetic diseases. Because mutated proteins are produced in specific cells, there is a critical need to develop organ- and cell-specific delivery strategies to realize the…

Intestinal mucosal wound resealing

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

ASMA NUSRAT · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2022 · $460,345 — Abstract The gastrointestinal epithelium plays a central role in maintaining and coordinating mucosal homeostasis and immunity. Intestinal epithelial barrier compromise in mucosal wounds is seen in many pathologic states that encompass inflammatory bowel diseases, ischemia, mechanical injury and sur…

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

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Admire Dube · STELLENBOSCH UNIVERSITY · 2022 · $328,171 — 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…

Signal transduction mechanisms that mediate normal and pathologic angiogenesis

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Jinjiang Pang · UNIVERSITY OF ROCHESTER · 2022 · $466,903 — 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…

Ultrasmall particle-based solutions for inducing ferroptosis and improving anti-tumor immune responses in cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2022

Michelle S Bradbury · SLOAN-KETTERING INST CAN RESEARCH · 2022 · $635,854 — Project Summary: Enormous strides continue to be made in the design of nanoparticles as highly specialized therapeutics for achieving superior outcomes over standard pharmacological agents, the latter often associated with significant toxicity that limits treatment efficacy. While cancer immunothera…

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 / 2022

S. Munir ALAM · DUKE UNIVERSITY · 2022 · $293,911 — 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…