Project Grant R01FD003432

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 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 $613,558 from the National Institute of Diabetes and Digestive and Kidney Diseases (CFDA 93.847 Diabetes, Digestive, and Kidney Diseases Extramural Research) supports Genetobe Inc., a Michigan-based biotechnology company, in developing novel, selective sodium-dependent glucose cotransporter 1 (SGLT1) inhibitor compounds as an adjunctive therapy for cystic fibrosis (CF) associated metabolic disorders, including CF-associated liver disease and CF-related diabetes. The...

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 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...

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 $812,621 Project Grant award, funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847), supports research to decipher the role of the cystic fibrosis transmembrane receptor (CFTR) in dietary fatty acid transport within the small intestine. The Principal Investigator is Alison B. Kohan, with Co-Principal Investigator CA Hodges. The project aims to use novel surgical...

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 $174,302 Project Grant awarded by the National Human Genome Research Institute (CFDA 93.172 - Human Genome Research) aims to characterize how adults with the childhood-onset genetic disease cystic fibrosis conceptualize their futures and plan their lives amid evolving prognoses and therapeutic innovations. The 5-year grant, awarded to the Trustees of the University of Pennsylvania, will use a disability studies framework to conduct qualitative interviews with adults with cystic fibrosis...