Creating tools to improve gene editing techniques for treating genetic disorders
Official title: Development of tools for rapid systematic refinement of in vivo gene editing technologies
Funded at Indiana University Indianapolis · NIH-10740025 · Data updated 2026-11-12
This study is working on improving gene editing tools to help fix genetic disorders by using special viruses to deliver the editing components, which could lead to better and safer treatments for patients with these conditions.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Indiana University Indianapolis NIH-funded |
| Lab location | 1 site (Indianapolis, United States) |
| Project start | August 15, 2023 |
| Project end | August 14, 2025 |
| Funding year | 2023 |
| Award (this year) | $425,348 |
| NIH project number | 1R21HD108460-01A1 |
| Project ID | NIH-10740025 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders that could potentially be treated through gene editing technologies.
✕Less likely to help
Patients with non-genetic disorders or those whose conditions do not involve gene editing may not benefit from this research.
What this research studies
This research focuses on enhancing the precision and effectiveness of gene editing technologies, specifically using adeno-associated viruses (AAV) to deliver CRISPR/Cas9 components for correcting genetic defects. By developing high-throughput methods to analyze and optimize the efficiency of AAV-mediated gene editing in living organisms, the research aims to better understand the mechanisms involved and improve safety for potential clinical applications. Patients may benefit from more effective treatments for genetic disorders as these tools are refined and validated.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with genetic disorders.
How similar studies have gone: Previous research has shown promising results with CRISPR/Cas9 and AAV technologies, indicating potential for success in this novel approach.
Where this research is happening
-
Indiana University Indianapolis
Indianapolis, United States Active
Researchers
- Principal investigator: Vandusen, Nathan James — Indiana University Indianapolis
- Study coordinator: Vandusen, Nathan James
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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.