New methods to deliver gene editing tools for treating genetic diseases
Nonviral delivery techniques for in vivo prime editing
This study is exploring new ways to deliver a promising gene editing technique that could fix genetic mutations causing diseases, using tiny fat particles to safely send the treatment to the right parts of the body, which could lead to better and safer options for people with genetic disorders.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts Institute of Technology NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-11007263 on NIH RePORTER |
What this research studies
This research focuses on developing innovative delivery techniques for a cutting-edge gene editing method known as prime editing, which aims to correct genetic mutations responsible for diseases. The approach involves using lipid nanoparticles (LNPs) to safely and effectively transport prime editing components to targeted organs in the body. By employing combinatorial chemistry, the researchers will create a diverse library of biodegradable lipids and utilize a novel screening method called pegRNA barcoding to identify the most effective LNP formulations in living models. This could lead to more efficient and safer treatments for genetic disorders.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that could be targeted by prime editing techniques.
Not a fit: Patients with non-genetic conditions or those whose genetic mutations are not amenable to prime editing may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a more effective and safer way to treat or potentially cure genetic diseases.
How similar studies have performed: Previous research has shown promise in gene editing techniques, but the specific combination of methods proposed here is novel and untested.
Where this research is happening
Cambridge, United States
- Massachusetts Institute of Technology — Cambridge, United States (Active)
Researchers
- Principal investigator: Anderson, Daniel G — Massachusetts Institute of Technology
- Study coordinator: Anderson, Daniel G
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.