A new method for delivering gene therapy to treat Cystic Fibrosis and other diseases
A non-viral nanoparticle gene therapy delivery platform for treating Cystic Fibrosis and other diseases
This study is working on a new way to deliver gene therapy for Cystic Fibrosis using tiny particles that don’t involve viruses, making it safer and potentially more effective for patients with CF and other lung diseases.
Quick facts
| Grant type | Sbir 1 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ogb5, INC. NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project ID | NIH-10915092 on NIH RePORTER |
What this research studies
This research focuses on developing a non-viral nanoparticle platform for delivering gene therapy specifically aimed at treating Cystic Fibrosis (CF) and potentially other chronic lung diseases. The approach utilizes a unique DNA and RNA nanoparticle technology that can carry large genes and includes enhancers to improve gene transfer efficiency to targeted cells. By overcoming the limitations of traditional viral delivery systems, this method aims to provide a safer and more effective way to deliver therapeutic genes directly to the lungs of patients. The research will involve both laboratory experiments and tests in animal models to evaluate the effectiveness of this delivery system.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Cystic Fibrosis who may benefit from advanced gene therapy treatments.
Not a fit: Patients with non-genetic lung diseases or those who do not have Cystic Fibrosis may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with Cystic Fibrosis, improving their lung function and overall quality of life.
How similar studies have performed: Previous research has shown promise in using non-viral delivery systems for gene therapy, indicating potential for success with this novel approach.
Where this research is happening
Cincinnati, United States
- Ogb5, INC. — Cincinnati, United States (Active)
Researchers
- Principal investigator: Sheehan, Colin T. — Ogb5, INC.
- Study coordinator: Sheehan, Colin T.
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.