Project Grant R01HL180559
- 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...
- 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...
- 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 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...
- 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 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, totaling $756,875.00, was provided by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) to the University of Cincinnati. The award supports research to investigate the fundamental mechanisms by which neutrophil extracellular traps (NETs) contribute to airway damage and infection in mucoobstructive lung diseases, such as cystic fibrosis. Key research aims include examining how NET-mediated...
- 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)...
- This Project Grant award from the National Heart Lung and Blood Institute (CFDA 93.837 - Cardiovascular Diseases Research) provides funding of $374,576 from September 5, 2025 to August 31, 2027 to Louisiana State University Health Sciences Center New Orleans. The grant supports research to investigate the role of neutrophils, a type of immune cell, in the pathogenesis of cystic fibrosis (CF). The research aims to elucidate how CFTR, the gene mutated in CF, impacts neutrophil function and the...
- 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...
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 modulator therapies. The goal is to identify the cellular mechanisms and real-world impacts of the SLC26A9 gene on CFTR modulator response, which could inform future personalized treatment strategies and CFTR-focused gene therapies for CF. The project will run from August 2025 through May 2030.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $727.6k | 8/15/25 |