Developing a gene editing therapy for cystic fibrosis using airway stem cells
A universal genome editing strategy to develop an airway stem cell therapy for cystic fibrosis
This study is exploring a new way to help people with cystic fibrosis by using gene editing to fix the genetic problems in airway stem cells taken from the sinuses, with the hope of improving lung function and reducing infections.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Research Inst Nationwide Children's Hosp NIH-funded |
| Lab location | 1 site (Columbus, United States) |
| Project ID | NIH-10909248 on NIH RePORTER |
What this research studies
This research focuses on creating a new therapy for cystic fibrosis (CF) by using gene editing techniques to correct mutations in airway stem cells. The approach involves extracting stem cells from the sinuses, where they are more accessible, and using advanced gene editing tools to fix the genetic defects that cause CF. By correcting these mutations, the goal is to improve lung function and reduce the frequency of respiratory infections in patients. This innovative method aims to provide a more effective and lasting treatment option for individuals with CF.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals diagnosed with cystic fibrosis, particularly those with the common F508del mutation.
Not a fit: Patients with cystic fibrosis who do not have genetic mutations that can be corrected by this gene editing approach may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking therapy that significantly improves lung health and quality of life for cystic fibrosis patients.
How similar studies have performed: While gene therapy for cystic fibrosis has been attempted in the past, this specific approach using sinus-derived stem cells and advanced gene editing techniques is novel and has not been widely tested.
Where this research is happening
Columbus, United States
- Research Inst Nationwide Children's Hosp — Columbus, United States (Active)
Researchers
- Principal investigator: Vaidyanathan, Sriram — Research Inst Nationwide Children's Hosp
- Study coordinator: Vaidyanathan, Sriram
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 below.