A5091745450- Cystic Fibrosis Research Advances
- Tissue Engineering and Regenerative Medicine
- Pharmaceutical studies and practices
- Neonatal Respiratory Health Research
- RNA Interference and Gene Delivery
- Advanced biosensing and bioanalysis techniques
Yale University
2022-2024
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. We sought to correct multiple organ dysfunction of F508del CF-causing mutation using systemic delivery peptide nucleic acid gene editing technology mediated biocompatible polymeric nanoparticles. confirmed phenotypic and genotypic modification vitro primary nasal epithelial cells from mice grown at air-liquid interface vivo following intravenous delivery. In treatment resulted a partial...
The CFTR modulator Trikafta has markedly improved lung disease for Cystic Fibrosis (CF) patients carrying the common delta F508 (F508del-CFTR) mutation. F508del-CFTR results in an apical trafficking defect and loss of function CFTR-expressing epithelial cells. However, not resulted gastrointestinal CF patients. A humanized mouse model was recently generated to evaluate modulators other compounds treat human intestinal disease. Short-term (4h) treatment rats with Dexamethasone (Dex) potently...