Using advanced gene editing to insert functional genes for cystic fibrosis treatment
Leveraging CRISPR RNA-guided DNA Transposases for Gene Insertion at the CFTR Locus
['FUNDING_FELLOWSHIP'] · COLUMBIA UNIVERSITY HEALTH SCIENCES · NIH-11005254
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
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | COLUMBIA UNIVERSITY HEALTH SCIENCES (nih funded) |
| Locations | 1 site (NEW YORK, UNITED STATES) |
| Trial ID | NIH-11005254 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with cystic fibrosis, particularly those with various mutations in the CFTR gene.
Not a fit: Patients without cystic fibrosis or those whose conditions are not related to CFTR gene mutations may not benefit from this research.
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 performed: 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
NEW YORK, UNITED STATES
- 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 below.
Conditions: Candidate Disease Gene