Creating safer and more effective tools for gene editing using CRISPR technology
Development of potent and safe CRISPR tools for in vivo gene editing using directed evolution
['FUNDING_R21'] · SCRIPPS RESEARCH INSTITUTE, THE · NIH-11083776
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
| Phase | ['FUNDING_R21'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | SCRIPPS RESEARCH INSTITUTE, THE (nih funded) |
| Locations | 1 site (LA JOLLA, UNITED STATES) |
| Trial ID | NIH-11083776 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals suffering from genetic diseases, blood disorders, or certain types of cancer.
Not a fit: Patients with non-genetic conditions or those who do not have access to gene editing therapies may not benefit from this research.
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 performed: 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
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.
Conditions: Blood Diseases, blood disorder, Cancers