Safer, stronger CRISPR tools for gene therapies
Development of potent and safe CRISPR tools for in vivo gene editing using directed evolution
Developing improved CRISPR gene-editing methods to make future treatments safer and more effective for people with inherited blood disorders.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Scripps Research Institute, the NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-11326711 on NIH RePORTER |
What this research studies
This project works to create more powerful and safer CRISPR gene-editing tools that could be used inside the body. Researchers will use directed evolution in the lab to refine the editing proteins and will chemically modify the guide RNAs to make them more stable and less likely to trigger immune reactions. The work is largely laboratory-based, using cell models and preclinical systems to test potency, specificity, and safety. If the tools perform well in these tests, they could move toward animal studies and eventually clinical trials in patients.
Who could benefit from this research
Good fit: People with inherited genetic blood disorders that might be treated by in-body gene editing would be the likely candidates for future trials based on this work.
Not a fit: Patients whose conditions are not caused by a single correctable gene defect or whose care does not involve gene editing are unlikely to benefit from this work in the near term.
Why it matters
Potential benefit: Could enable permanent, safer gene-correcting treatments for certain inherited blood diseases by reducing toxicity and improving editing precision.
How similar studies have performed: CRISPR-based therapies have shown early clinical successes for some genetic diseases, but combining directed evolution with full chemical modification of guide RNAs is a newer laboratory approach still under testing.
Where this research is happening
La Jolla, United States
- Scripps Research Institute, the — La Jolla, United States (Active)
Researchers
- Principal investigator: Miller, Shannon Marie — Scripps Research Institute, the
- Study coordinator: Miller, Shannon Marie
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.