Project Grant R43HL170830
- This Project Grant award of $520,483 from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) supports research at The University of Texas Southwestern Medical Center to develop adeno-associated virus (AAV)-based gene therapy tools for efficient delivery of therapeutic cargos to the atrioventricular node (AVN) of the cardiac conduction system. The long-term goal is to devise new therapeutic approaches for cardiac dysrhythmias. The key objectives are to...
- This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI) under CFDA 93.837 - Cardiovascular Diseases Research provides $704,635 to the University of Florida to develop optimized adeno-associated virus (AAV) vectors for gene therapy of hemophilia. Specifically, the award aims to engineer both capsid- and genome-modified AAV3 vectors that can transduce human hepatocytes more efficiently than current AAV5 vectors, potentially reducing the required vector dose and...
- This federal Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), part of the Cardiovascular Diseases Research program (CFDA 93.837), provides $300,000 to Gigamune, Inc. to develop an in vivo gene therapy for sickle cell disease. The project aims to test a novel lentiviral vector technology for efficiently editing hematopoietic stem cells to knockout the BCL11A enhancer, a key genetic target for sickle cell treatment. Through in vitro assays, the Phase I effort will...
- This federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) provides $611,014 to the University of Connecticut Health Center to develop and optimize adeno-associated virus (AAV) therapies for treating cardiac disorders caused by pathogenic variants in the TNNT2 gene. The project aims to engineer next-generation AAVs capable of delivering wild-type TNNT2 and selectively suppressing mutant TNNT2 mRNA in cardiomyocytes. This...
- The National Center for Advancing Translational Sciences (NCATS), under CFDA Program 93.350, has awarded a $331,034 Project Grant to Vector Surgical LLC to develop the Gene Therapy Catheter (GTC) - an electrical pulsing device designed to enhance the delivery of adeno-associated virus (AAV) gene therapy to the liver. The GTC aims to improve the efficiency and safety of liver-directed gene therapies by combining local delivery with electrical pulsing and a double-balloon system to isolate the...
- This Project Grant award for $314,363, provided by the National Heart, Lung, and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel gene therapy technology called STITCHR to enable full gene replacement for Hemophilia A. The proposed research will engineer robust adeno-associated virus (AAV) constructs to deliver and express the full-length Factor VIII gene, addressing the limitations of current truncated versions. The project will...
- The National Heart, Lung, and Blood Institute (NHLBI) awarded a $243,000 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to the University of Cincinnati's Sponsored Research Services Division. The grant will fund a 2-year project to engineer advanced RNA biodevices that leverage CRISPR and ADAR gene-editing technologies. The goal is to improve the efficiency and specificity of converting resident cardiac fibroblasts into functional cardiomyocytes, in order to boost...
- This Project Grant award, valued at $667,704.00, was provided by the National Heart Lung and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837) to address hypertrophic cardiomyopathy (HCM), a genetic heart condition affecting 1 in 500 adults. The primary objective is to develop chemically inducible split base editors (SBE) that can precisely and controllably edit the MYBPC3 gene in the heart to correct mutations that cause HCM. The research aims to expand...
- This Project Grant award from the Trans-NIH Research Support program, CFDA #93.310, provides $1,350,770 to The Leland Stanford Junior University to develop a novel class of synthetic RNA-based receptors to enhance the targeting precision of mRNA-mediated adoptive cell therapies. The project aims to create a modular, programmable receptor system that can convert ligand-induced dimerization events into the expression of desired output proteins, enabling improved specificity for ablating target...
- This Project Grant award of $786,188, provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop a novel gene therapy platform to prevent the failure of prosthetic bypass grafts used to treat arterial blockages in the lower extremities. The research, conducted by Beth Israel Deaconess Medical Center, Inc. (BIDMC), focuses on optimizing a biodegradable hydrogel system that can deliver small interfering RNA...
APTAMER ENGINEERING OF LENTIVIRAL VECTORS FOR CARDIAC GENE THERAPIES - PROJECT SUMMARY/ABSTRACT OVERVIEW: HYPERTROPHIC CARDIOMYOPATHY IS THE LEADING CAUSE OF SUDDEN DEATH IN YOUNG INDIVIDUALS, WHERE SEVERAL LOSS-OF-FUNCTION MUTATIONS IN KEY SARCOMERIC PROTEINS CAUSE HYPERTROPHY. GENE THERAPY APPROACHES THAT DRIVE THE OVEREXPRESSION OF THE CORRECT GENES CAN BE USED TO REMODEL THE HEART TISSUE AND RESTORE FUNCTION. HOWEVER, NO GENE THERAPIES HAVE BEEN DEVELOPED FOR IN VIVO TARGETING OF THE HEART TO DATE. A KEY BOTTLENECK IN THE FIELD IS THE ABILITY TO ENGINEER ADVANCED VIRAL VECTORS WITH PRESCRIBED TROPISM IN THE PATIENT'S BODY. LENTIVIRAL VECTOR (LVV) ENGINEERING APPLIES RATIONAL DESIGN PRINCIPLES TO DEFINE COMPONENTS OF THE VECTOR ENVELOPE, SUCH AS ATTACHMENT PROTEINS, LEADING TO VECTORS WITH PROGRAMMABLE TISSUE SPECIFICITY AND OFF-TARGET ACTIVITY.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $0 | 11/25/24 | ||
| Not listed | $289.7k | 8/11/23 |