AIR-LIPID-CF: Defining an Airway Lipidomic Readiness Panel in Cystic Fibrosis for Future Gene and Modulator Therapies
ProjectScientific Basis for the Proposed Research
Thousands of people with cystic fibrosis (pwCF) still lack effective CFTR modulator therapy because of rare or non-responsive mutations. As gene and mRNA therapies advance, there is an urgent need for noninvasive, drug-agnostic biomarkers that reflect airway epithelial health and readiness for therapeutic benefit. Lipids and metabolites regulate membrane repair, vesicle trafficking, and inflammation resolution. Nasal airway swabs (NAS) sampling provides a minimally invasive approach to isolate extracellular vesicles (EVs) and soluble metabolites released from airway epithelial cells, offering a practical, repeatable window into mucosal biology. EVs carry bioactive lipids and metabolites that can mirror epithelial and immune status. Preliminary observations from our group show distinct lipidomic patterns in CF airway samples, supporting nasal EV profiling as a functional readout of epithelial readiness for next-generation therapies.
Thousands of people with cystic fibrosis (pwCF) still lack effective CFTR modulator therapy because of rare or non-responsive mutations. As gene and mRNA therapies advance, there is an urgent need for noninvasive, drug-agnostic biomarkers that reflect airway epithelial health and readiness for therapeutic benefit. Lipids and metabolites regulate membrane repair, vesicle trafficking, and inflammation resolution. Nasal airway swabs (NAS) sampling provides a minimally invasive approach to isolate extracellular vesicles (EVs) and soluble metabolites released from airway epithelial cells, offering a practical, repeatable window into mucosal biology. EVs carry bioactive lipids and metabolites that can mirror epithelial and immune status. Preliminary observations from our group show distinct lipidomic patterns in CF airway samples, supporting nasal EV profiling as a functional readout of epithelial readiness for next-generation therapies.