Using advanced gene editing to insert functional genes for cystic fibrosis treatment
Official title: Leveraging CRISPR RNA-guided DNA Transposases for Gene Insertion at the CFTR Locus
Funded at Columbia University Health Sciences · NIH-11005254 · Data updated 2026-11-12
This study is testing a new way to safely fix the gene that causes cystic fibrosis, which could help many patients by providing a more reliable treatment option without the usual risks.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Columbia University Health Sciences NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | February 1, 2023 |
| Project end | January 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $53,974 |
| NIH project number | 5F31HL167530-03 |
| Project ID | NIH-11005254 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with various mutations in the CFTR gene.
✕Less likely to help
Patients without cystic fibrosis or those whose conditions are not related to CFTR gene mutations may not benefit from this research.
What this research studies
This research focuses on developing a new gene editing technology that allows for the precise insertion of large DNA sequences without causing harmful double-strand breaks in the DNA. By utilizing CRISPR-associated transposons, the project aims to create a method that can insert a functional copy of the CFTR gene directly into the genome of patients with cystic fibrosis, regardless of their specific genetic mutations. The approach seeks to enhance the safety and effectiveness of gene therapy for cystic fibrosis, potentially leading to a universal treatment option. Patients may benefit from this innovative technique as it could provide a more reliable and less risky way to correct the underlying genetic defects causing their condition.
Why it matters
Potential benefit: If successful, this research could lead to a universal cure for cystic fibrosis by enabling the insertion of a functional CFTR gene in patients' cells.
How similar studies have gone: Other research has shown promise in using CRISPR technology for gene editing, but this specific approach utilizing RNA-guided transposases is relatively novel and untested.
Where this research is happening
-
Columbia University Health Sciences
New York, United States Active
Researchers
- Principal investigator: King, Rebeca Teresa — Columbia University Health Sciences
- Study coordinator: King, Rebeca Teresa
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.