New gene editing methods for treating cystic fibrosis
Official title: Development of novel approaches for gene editing therapies of cystic fibrosis
Funded at University of Kansas Medical Center · NIH-11166571 · Data updated 2026-11-12
This study is exploring a new way to use gene editing to help people with cystic fibrosis by fixing the faulty gene that causes the condition, aiming to create a lasting treatment that works better for lung cells.
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 | 2025 |
| Award (this year) | $561,071 |
| NIH project number | 5R01HL174593-02 |
| Project ID | NIH-11166571 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with cystic fibrosis, particularly those with mutations in the CFTR gene who are currently not benefiting from existing CFTR modulator therapies.
✕Less likely to help
Patients with cystic fibrosis who have already undergone successful lung transplants or those with other unrelated severe health conditions may not benefit from this research.
What this research studies
This research focuses on developing innovative gene editing therapies aimed at treating cystic fibrosis (CF), a genetic disorder caused by mutations in the CFTR gene. The approach involves using CRISPR-based techniques to correct these mutations, specifically targeting airway progenitor stem cells to ensure effective and regulated CFTR expression. By utilizing a novel method called homology-independent targeted integration (HITI), the research aims to improve the efficiency of gene correction in lung cells, which are often difficult to treat with traditional methods. This could potentially provide a more permanent solution for patients with various CFTR mutations.
Why it matters
Potential benefit: If successful, this research could lead to a more effective and less costly treatment option for cystic fibrosis patients, potentially offering a cure for all CFTR genotypes.
How similar studies have gone: Previous research has shown promise in using gene editing techniques for genetic disorders, indicating that this approach could lead to significant advancements in cystic fibrosis treatment.
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.