Developing advanced gene editing techniques for therapeutic use
Gene Editing Core
This study is working on improving gene editing techniques to make treatments for genetic conditions safer and more effective, which could help patients get better care.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Jackson Laboratory NIH-funded |
| Lab location | 1 site (Bar Harbor, United States) |
| Project ID | NIH-11089346 on NIH RePORTER |
What this research studies
This research focuses on creating and optimizing gene editing strategies to improve therapeutic outcomes while reducing unwanted effects. The Gene Editing Core at the Broad Institute of MIT and Harvard will assess the effectiveness of these techniques on isolated cell lines and animal tissues. By analyzing both on-target and off-target editing activities, the project aims to refine gene editing technologies for specific therapeutic targets. Patients may benefit from the advancements in gene editing that could lead to more effective treatments for various genetic conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic conditions that may be addressed through gene editing technologies.
Not a fit: Patients with conditions that are not amenable to gene editing or those who do not have a genetic basis for their condition may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more precise and effective gene therapies for patients with genetic disorders.
How similar studies have performed: Previous research in gene editing has shown promising results, indicating that this approach has the potential for significant advancements in treatment.
Where this research is happening
Bar Harbor, United States
- Jackson Laboratory — Bar Harbor, United States (Active)
Researchers
- Principal investigator: Liu, David R — Jackson Laboratory
- Study coordinator: Liu, David R
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.