Investigating genetic mutations in lung diseases using advanced editing techniques
Using base editing to investigate CFTR polymorphisms in lung diseases
['FUNDING_OTHER'] · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · NIH-11092296
This study is looking at how changes in a specific gene can affect lung diseases like cystic fibrosis and asthma, and it aims to fix these changes in lung cells to help improve treatments for people living with these conditions.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11092296 on ClinicalTrials.gov |
What this research studies
This research focuses on understanding how genetic mutations in the CFTR gene contribute to various lung diseases, including cystic fibrosis and asthma. By utilizing a technique called base editing, the researchers aim to correct harmful mutations in lung cells and explore the effects of non-harmful genetic variations on lung function. Patients' primary lung cells will be used to test the effectiveness of these corrections, with the goal of improving treatment options for those affected by these conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with cystic fibrosis or those at risk for lung diseases associated with CFTR gene mutations.
Not a fit: Patients without CFTR mutations or those with unrelated lung conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that effectively treat or prevent lung diseases linked to CFTR mutations.
How similar studies have performed: Previous research has shown promising results using similar gene-editing techniques to correct genetic mutations, indicating potential for success in this approach.
Where this research is happening
NEW YORK, UNITED STATES
- ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI — NEW YORK, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: JIANG, TINGTING — ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
- Study coordinator: JIANG, TINGTING
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.