Creating safer and more effective tools for gene editing using CRISPR technology
Official title: Development of potent and safe CRISPR tools for in vivo gene editing using directed evolution
Funded at Scripps Research Institute, the · NIH-11083776 · Data updated 2026-11-12
This study is working on improving gene editing tools to make them safer and more effective for treating genetic diseases, so patients can have better and more reliable treatment options.
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 start | May 3, 2024 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $230,000 |
| NIH project number | 5R21EB036298-02 |
| Project ID | NIH-11083776 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals suffering from genetic diseases, blood disorders, or certain types of cancer.
✕Less likely to help
Patients with non-genetic conditions or those who do not have access to gene editing therapies may not benefit from this research.
What this research studies
This research focuses on developing advanced CRISPR tools for gene editing that are both potent and safe for use in living organisms. By utilizing directed evolution, the researchers aim to enhance the effectiveness of CRISPR-Cas9 technology while minimizing potential side effects. The approach involves modifying RNA components to improve their stability and reduce toxicity, which is crucial for successful gene therapy applications. Patients with genetic diseases could benefit from these innovations as they may lead to more reliable and safer treatment options.
Why it matters
Potential benefit: If successful, this research could provide patients with safer and more effective gene therapies for genetic disorders and cancers.
How similar studies have gone: Other research has shown promising results with CRISPR technology in clinical trials, indicating that this approach has the potential for significant advancements in gene therapy.
Where this research is happening
-
Scripps Research Institute, the
La Jolla, United States Active
Researchers
- Principal investigator: Miller, Shannon Marie — Scripps Research Institute, the
- Study coordinator: Miller, Shannon Marie
Related research
Other NIH-funded projects on the same topics.
-
The Participant Center helps people join and stay in a nationwide health study
A nationwide program to support people in joining, contributing biosamples, and staying connected to a large health study in the United States.
-
Data Sharing and Bioinformatics Core for Viral Systems Biology
A center-led effort to manage, share, and interpret large viral systems biology data using open tools and a community-friendly portal.
-
Germline-targeted HIV vaccines using membrane liposome tech to trigger bnAbs
A new HIV vaccine approach aims to spark broadly neutralizing antibodies by showing MPER on membranes, tested in models and early studies to guide future human trials.
-
Antivirals for hemorrhagic fever viruses like LASV, SFTSV, and EBOV
A multi-institutional effort to find and validate antiviral compounds that block key viral proteins from LASV, SFTSV, and EBOV to help people at risk from serious hemorrhagic fevers.
-
Developing new antivirals for flaviviruses
A research team is working to create oral antiviral drugs targeting flaviviruses to treat infections that affect millions of people.
-
CoV inhibitors targeting non-enzymatic SARS-CoV-2 components
A multicenter research effort seeks antiviral ideas that block SARS-CoV-2 parts that don’t work through traditional enzymes, aiming to help people affected by COVID-19.
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.