Gene-editing approaches to correct the CFTR gene in cystic fibrosis
Official title: Development of novel approaches for gene editing therapies of cystic fibrosis
Funded at University of Kansas Medical Center · NIH-11306696 · Data updated 2026-11-12
New gene-editing methods aim to permanently fix the CFTR gene for people with cystic fibrosis.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project start | August 10, 2024 |
| Project end | April 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $605,879 |
| NIH project number | 5R01HL174593-03 |
| Project ID | NIH-11306696 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with cystic fibrosis of any CFTR genotype—especially those who produce little or no CFTR protein and do not benefit from current modulators—would be the eventual candidates.
✕Less likely to help
Patients with very advanced lung disease, those unable to undergo airway delivery procedures, or those needing immediate symptom relief are unlikely to benefit during these early-stage efforts.
What this research studies
Researchers are developing CRISPR-based gene-editing techniques that use a method called HITI to insert a correct CFTR gene into airway cells, including quiescent airway stem cells. They plan to deliver the corrective DNA using viral tools such as AAV vectors so the edit can work in non-dividing cells that maintain the lung lining. The team is focused on restoring normal patterns of CFTR expression across different airway cell types rather than simply adding unregulated CFTR. Most activities described are preclinical laboratory and animal work to show the approach can safely and permanently correct the defect.
Why it matters
Potential benefit: If successful, this approach could provide a one-time genetic correction that restores normal CFTR function across many CFTR mutation types.
How similar studies have gone: CRISPR-based editing and HITI have shown promise in lab and animal models for correcting genes in non-dividing cells, but a proven, permanent CRISPR cure for CF in humans has not yet been achieved.
Where this research is happening
-
University of Kansas Medical Center
Kansas City, United States Active
Researchers
- Principal investigator: Qiu, Jianming — University of Kansas Medical Center
- Study coordinator: Qiu, Jianming
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.