Nonviral lipid delivery for prime gene editing in the body
Nonviral delivery techniques for in vivo prime editing
This project works to deliver prime editing tools into organs using nonviral lipid nanoparticles to fix genetic mutations for people with conditions like cystic fibrosis.
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-11258491 on NIH RePORTER |
What this research studies
Researchers will create large libraries of biodegradable lipids and build them into lipid nanoparticle (LNP) formulations. They will use a new pegRNA barcoding method that lets them test many different LNPs at once inside mice to see which ones deliver prime editors best to disease-relevant tissues. The approach combines combinatorial chemistry to make many candidate lipids with a high-throughput in vivo screen to find safe, potent delivery vehicles. Successful LNPs from this work could be advanced toward studies that eventually include people.
Who could benefit from this research
Good fit: Although the current work is preclinical in mice, eventual human trials would focus on people with genetic diseases caused by specific mutations, such as certain forms of cystic fibrosis.
Not a fit: Patients without mutations addressable by prime editing or those seeking immediate treatments are unlikely to benefit from this preclinical work in the near term.
Why it matters
Potential benefit: If successful, this could enable safer, nonviral delivery of prime editing to correct disease-causing mutations and move toward long-lasting treatments or cures.
How similar studies have performed: Lipid nanoparticle delivery has worked well for mRNA vaccines and some gene-editing approaches, but combining LNPs with prime editing and high-throughput in vivo barcoded screens is a newer strategy with limited prior clinical success.
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.