Project Grant R21HL179437
- The federal Project Grant award of $730,268.00 was provided by the National Heart Lung and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837) to the Icahn School of Medicine at Mount Sinai. The funding will support the development of a versatile paired prime editing toolkit to correct mutations in the CFTR gene, which is the primary cause of cystic fibrosis and associated with other genetic diseases. The research aims to replace defective CFTR exons with corrected...
- The Project Grant award of $564,121 from the National Heart Lung and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) will support research to develop peptide surface-functionalized lipid nanoparticles (PepLNPs) for pulmonary delivery that can reach airway basal cells and use base editing technology to correct specific CFTR gene mutations associated with cystic fibrosis. The goal is to achieve permanent correction of lung disease in cystic fibrosis patients through one-time,...
- This Project Grant award of $235,500 from the National Institute of Biomedical Imaging and Bioengineering (NIBIB) under the Discovery and Applied Research for Technological Innovations to Improve Human Health program (CFDA 93.286) supports the development of a microscale robotic system to facilitate adeno-associated virus serotype 4 (AAV4)-mediated gene delivery of the cystic fibrosis transmembrane conductance regulator (CFTR) to the small airway epithelium in cystic fibrosis patients. The...
- This Project Grant award from the National Heart Lung and Blood Institute, under the Cardiovascular Diseases Research federal grant program (CFDA 93.837), aims to develop a gene editing strategy to correct the CFTR-G542X mutation, the second most common and most prevalent null mutation causing cystic fibrosis (CF). The $129,639 grant, awarded on June 18, 2025, will support research to leverage Type I CRISPR base editing to install a precise single base change and correct the CFTR gene in...
- This Project Grant award of $629,319.00 from the National Heart Lung and Blood Institute (CFDA #93.837 Cardiovascular Diseases Research) supports research by Emory University to investigate cellular mechanisms for overcoming refractory cystic fibrosis defects. The research aims to identify ribosomal proteins that can partially rescue the synthesis and assembly of cystic fibrosis transmembrane conductance regulator (CFTR) protein variants with premature termination codons, which are associated...
- This Project Grant award of $727,575.00 from the National Heart, Lung, and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) aims to investigate the impact of the SLC26A9 gene on response to CFTR correction therapies for cystic fibrosis (CF) patients. The award will fund research at Children's Hospital Medical Center in Cincinnati, Ohio to evaluate how the SLC26A9 genotype affects lung function and respiratory epithelium physiology in CF patients with varying responses to CFTR...
- This federal Project Grant award, worth $809,162.00, was provided by the National Heart Lung and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837) to The Children's Hospital Corporation, doing business as Boston Children's Hospital, located in Massachusetts. The funding supports research to elucidate the differentiation pathways and functions of pulmonary ionocytes, a rare cell type in the human airway that expresses high levels of the cystic fibrosis transmembrane...
- The National Heart, Lung, and Blood Institute (NHLBI) awarded a $299,995 Project Grant under the Cardiovascular Diseases Research (CFDA 93.837) program to Nanite Inc., a small disadvantaged business in Massachusetts. The grant will fund the development of computationally-designed polymer-based delivery vehicles for mRNA payloads to treat cystic fibrosis (CF), a debilitating genetic disorder affecting the lungs. The project aims to leverage computational modeling and high-throughput polymer...
- This Project Grant awarded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) provides $750,672 to The Leland Stanford Junior University to conduct research on identifying the cellular protein quality control (PQC) machinery that underlies treatment resistance in cystic fibrosis (CF) patients. The objectives are to: 1) comprehensively identify genes that govern the stability of the most common CF-causing variant (F508del)...
- The federal Project Grant award from the National Heart, Lung, and Blood Institute (CFDA 93.837 Cardiovascular Diseases Research) supports research to understand the mechanisms linking the cystic fibrosis transmembrane conductance regulator (CFTR) gene to dysregulated barrier function, insulin receptor function, and glucose transport in the lungs of cystic fibrosis (CF) patients. The $730,494 award to Emory University, with a sub-award to Vanderbilt University Medical Center, aims to investigate...
This federal Project Grant award of $441,375.00, provided by the National Heart Lung and Blood Institute under the Cardiovascular Diseases Research program (CFDA 93.837), aims to develop and optimize a dual adeno-associated virus (AAV) system with intein technology to deliver full-length, functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to airway epithelial cells of cystic fibrosis (CF) patients. The goal is to restore CFTR function in CF patients, particularly those who do not respond to CFTR modulator therapies. The award recipient, Children's Hospital Medical Center (doing business as Children's Home Healthcare), will test this dual AAV-intein CFTR delivery approach in primary airway cells from CF patients and in a relevant mouse model to assess efficiency and durability. This research aims to advance a potential gene therapy solution for CF that could benefit patients unresponsive to current CFTR modulator treatments.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $441.4k | 8/27/25 |