CFF funds cell-based strategy to help rebuild damaged CF airways
Researchers will test gene-corrected airway cells and a biodegradable scaffold
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The Cystic Fibrosis Foundation is funding a new research project that aims to develop a cell-based strategy for rebuilding damaged airway tissue in people with cystic fibrosis (CF).
Do-Yeon Cho, MD, a professor in the Department of Otolaryngology–Head and Neck Surgery at the University of Alabama at Birmingham (UAB), received the Path to a Cure Pilot & Feasibility Award. The Cystic Fibrosis Foundation’s Path to a Cure initiative supports research aimed at developing transformative therapies and ultimately finding a cure for CF.
Cho will lead the project, titled “Evaluating Extracellular Matrix–Based Regeneration for Airway Repair Using CFTR-Targeted Tools in CF.”
Researchers target damaged airway tissue
“Our goal is to develop regenerative therapies that rebuild healthy airway tissue and restore the airway’s natural ability to defend itself against chronic infection,” Cho, who is also the section chief of otolaryngology at the Birmingham VA Health Care System, said in a UAB press release.
CF is caused by mutations in the CFTR gene, which provides instructions for making a protein that regulates the movement of salt and water across cell membranes.
When the CFTR protein is defective or absent, thick and sticky mucus accumulates in organs such as the lungs. This mucus is difficult to clear and can trap bacteria in the airways, contributing to repeated infections, inflammation, and progressive tissue damage.
CFTR modulators target specific defects in the CFTR protein and have substantially improved outcomes for many people with eligible mutations. However, chronic airway inflammation and tissue damage can continue to affect quality of life and long-term health.
“While current CFTR modulators have transformed the care of patients with cystic fibrosis, they cannot fully repair the airway damage that has already occurred,” Cho said.
The airway epithelium — the layer of cells lining the respiratory tract — helps clear mucus and defend the body against infection. Persistent damage to this lining can weaken those defenses and contribute to long-term respiratory problems.
Cho’s team will focus on airway basal cells, specialized stem-like cells that play a role in the maintenance and repair of the airway lining. The researchers will study whether CFTR defects can be corrected in these cells and whether the corrected cells can then be transplanted into damaged airways to help rebuild a healthier epithelial lining. A biodegradable scaffold will support the transplanted cells as they integrate with existing tissue.
Rabbit model will test regenerative strategy
A rabbit model of CF will be used to test the strategy. According to the press release, the model will allow the researchers to evaluate how well transplanted cells integrate into existing tissue, rebuild the airway lining, and restore normal airway function.
Ultimately, the researchers aim to establish a roadmap for combining gene correction, cell transplantation, and tissue engineering in future regenerative treatments for CF and other disorders involving airway injury.F
“If successful, this work could provide the foundation for a new generation of cell-based regenerative treatments, not only for cystic fibrosis but also for other chronic airway diseases where epithelial injury plays a central role,” Cho said.
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