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9873 documents

Targeting endolysosomal trafficking to increase delivery of antisense oligonucleotides to tumors

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Brendan Tyler Finicle · UNIVERSITY OF CALIFORNIA-IRVINE · 2021 · $18,445 — PROJECT SUMMARY/ABSTRACT Antisense oligonucleotides (ASO) are the ultimate platform technology that could cripple lethal, drug-resistant tumors by targeting “undruggable” oncogenes. ASO are 16-20 bp nuclease-resistant oligonucleotides that base pair with a target RNA and can elicit its degradation o…

Mapping Cancer Metabolism by Mid-infrared Photothermal Microscopy

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Ji-Xin Cheng · BOSTON UNIVERSITY (CHARLES RIVER CAMPUS) · 2021 · $398,440 — Program Summary While altered cell metabolism is emerging as a hallmark of cancer, there is an unmet need for new tools for quantitation of metabolites. NMR spectroscopy, mass spectrometry, FTIR, and Raman spectroscopy are widely used for molecular detection in tissue extracts or intact tissues. Ye…

Barrier Damage and The Immune CascadeNorthwestern University CounterACT Center of Excellence (NUCCX)

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

ROBERT M LAVKER · NORTHWESTERN UNIVERSITY AT CHICAGO · 2021 · $2,786,961 — Northwestern University (NU) is recognized for its strong interdisciplinary cutaneous, ocular, nanotechnology and immunology research programs with particular strength in investigations related to wound repair and inflammation. Leveraging these strengths, the theme of the NU CounterAct Center of Exc…

Controlled release of RNA-targeting therapy to promote healing of diabetic ulcers

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Adam G Berger · HARVARD MEDICAL SCHOOL · 2021 · $51,036 — Project Summary Non-healing ulcers are a common complication of diabetes, resulting in decreased quality of life, elevated rates of amputation, increased risk of mortality, and high healthcare costs. Unfortunately, current treatments remain outdated and inadequate. In diabetes, neuropathy and microv…

HIV-1 Detection and Elimination From CNS Mononuclear Phagocytes

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Howard E Gendelman · UNIVERSITY OF NEBRASKA MEDICAL CENTER · 2021 · $688,500 — 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 …

Molecular Genetics Of Heritable Human Disorders

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

JANICE CHOU · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · 2021 · $2,050,440 — The rAAV-co-G6PC vector used in the current phase I/II clinical trial is episomally expressed and the long-term durability of expression in humans is currently being established. We therefore sought to explore the use of the CRISPR/Cas9 technology to correct a pathogenic GSD-Ia variant in its native…

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

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

GABOR J TIGYI · UNIVERSITY OF MARYLAND BALTIMORE · 2021 · $301,081 — The US population at large, and particularly military personnel and first responders, are at risk of radiation exposure 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 th…

Signaling and Biotechology Program

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Hui-Kuan Lin · WAKE FOREST UNIVERSITY HEALTH SCIENCES · 2021 · $34,767 — The major goals of the newly formed Signaling and Biotechnology (SBT) Program are to understand the mechanisms underlying cancer pathogenesis and resistance to therapy and to use this knowledge to develop new technologies leading to more effective strategies for improved detection and treatment of c…

Molecular Imaging Using Cryo-EM

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Yifan Cheng · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · 2021 · $297,506 — As a versatile structure determination technology, cryo-EM is an integrated part of the HARC Center. In the past funding cycle, the Cryo-EM Core has engaged in technology development of single-particle cryo-EM, and has made major contributions to the recent technological breakthroughs in single part…

Intestinal mucosal wound resealing

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

ASMA NUSRAT · UNIVERSITY OF MICHIGAN AT ANN ARBOR · 2021 · $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…

Native Cell Membrane Nanoparticles System

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Youzhong Guo · VIRGINIA COMMONWEALTH UNIVERSITY · 2021 · $382,600 — SUMMARY Membrane proteins play crucial roles in living organisms. Protein-lipid interactions are crucial for the structural and functional integrity of membrane proteins. High-resolution of membrane protein structures are in high-demand for both understanding their biology and structure-based drug d…

ALCAM-mediated cell adhesion and extracellular vesicle biogenesis in bladder cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Ariana Kathryn von Lersner · VANDERBILT UNIVERSITY · 2021 · $30,754 — PROJECT SUMMARY Extracellular vesicles (EVs) are a heterogenous group of lipid bilayer enclosed particles secreted from cells and are found in most, if not all, biological fluids. The term EV encompasses a wide range of vesicle classes, including exosomes (50-150 nm) and microvesicles (150 nm - 100…

Continued Development and Maintenance of the UltraScan Software

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Emre H. Brookes · UNIVERSITY OF MONTANA · 2021 · $293,074 — Project Summary/Abstract We propose to continue development and support for the UltraScan-III (US3) software suite and develop new code and methods to address challenging biomedical research questions that can be solved with the latest instruments available for analytical ultracentrifugation and at …

Efficient in Vivo RNP-based Gene Editing in the Sensory Organ Inner Ear Using Bioreducible Lipid Nanoparticles

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Zheng-Yi Chen · MASSACHUSETTS EYE AND EAR INFIRMARY · 2021 · $1,638,192 — Project Summary and Relevance Application of genome editing based therapy requires efficient delivery of editing agents into disease-relevant tissues and cells. Identification of novel delivery materials targeting somatic cells will greatly facilitate the advance of editing based therapy strategies …

Nucleic acid modulators and theranostics for ADAR

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Guizhi Zhu · VIRGINIA COMMONWEALTH UNIVERSITY · 2021 · $388,125 — 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…

Programmable gene integration and cell engineering with CRISPR-directed integrases

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Omar O Abudayyeh · MASSACHUSETTS INSTITUTE OF TECHNOLOGY · 2021 · $568,123 — Project Summary Despite extraordinary advances in genome engineering, tools for precise and efficient gene correction across all cell types and desired edits remain lacking. Current programmable DNA cleavage tools, such as CRISPR-Cas9, rely on cellular DNA repair mechanisms, which are inefficient an…

Decoding Individual Exosomes in Cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

GREGORY W FARIS · NUMENTUS TECHNOLOGIES INC. · 2021 · $233,562 — PROJECT SUMMARY There is increasing recognition that extracellular vesicles (EVs)¾micrometer- or nanometer-sized lipid particles containing protein and nucleic acid cargoes (information)¾are highly promising for new diagnostics/prognostics and even have therapeutic value. There is, however, an unme…

Regulation of microRNA homeostasis: Implications in bone fracture healing

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Reyad A Elbarbary · PENNSYLVANIA STATE UNIV HERSHEY MED CTR · 2021 · $395,786 — Abstract Impaired or delayed fracture healing is a clinical problem that affects >1.5 million people in the US annually; obesity and associated type 2 diabetes (T2D) are significant and independent risk factors in this context. Nearly 34% of the US population is obese, and the number is projected to…

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

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Suchismita Acharya · UNIVERSITY OF NORTH TEXAS HLTH SCI CTR · 2021 · $358,091 — 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…

Using Targeted Lipid Nanoparticles to Deliver Chemotherapeutic Agents against Pancreatic Cancer

NIH Pandemic-Era Grants · Pandemic Era Grants / 2021

Maria Lambros · DORICPHARMA LLC · 2021 · $52,000 — PROJECT SUMMARY/ABSTRACT Pancreatic cancer continues to be a devastating disease with less than 10% 5-year survival. Pancreatic cancer is aggressive and there are no good tools for early detection. By the time of diagnosis, the tumor is often surgically unresectable. At this stage, patients are ofte…