Blocking a specific microRNA to improve treatment for cystic fibrosis
miR-145 target site blockade is a selective strategy to enhance CFTR restoration and readthrough
This study is looking at a new way to help kids with cystic fibrosis by blocking a tiny molecule that makes their treatment less effective, hoping to improve how well their current therapies work, especially for those who haven't had much success with them.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Alabama at Birmingham NIH-funded |
| Lab location | 1 site (Birmingham, United States) |
| Project ID | NIH-10900465 on NIH RePORTER |
What this research studies
This research focuses on enhancing the effectiveness of cystic fibrosis treatments by targeting a specific microRNA, miR-145, which negatively impacts the CFTR gene's function. The approach involves using an antisense oligonucleotide to selectively block miR-145 from binding to the CFTR gene, potentially improving the response to existing therapies. The research aims to investigate this strategy in children with cystic fibrosis, particularly those with specific genetic mutations that currently lack effective treatments. By manipulating miR-145, the researchers hope to enhance the correction of CFTR function in patients who are low responders to current therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are children under 11 years old who have cystic fibrosis, especially those with premature termination codon mutations or low responders to existing therapies.
Not a fit: Patients with cystic fibrosis who do not have the targeted genetic mutations or who are not under 11 years old may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to improved treatment options for children with cystic fibrosis, particularly those with specific genetic mutations.
How similar studies have performed: Other research has shown promise in using microRNA manipulation to enhance gene therapy outcomes, suggesting that this approach could be effective.
Where this research is happening
Birmingham, United States
- University of Alabama at Birmingham — Birmingham, United States (Active)
Researchers
- Principal investigator: Harris, William Thomas — University of Alabama at Birmingham
- Study coordinator: Harris, William Thomas
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.