Developing advanced methods for safe and effective gene editing therapies
Official title: Ultra-sensitive, unbiased, high-throughput, biochemical CHANGE-seq genome-wide activity and gRNA sequencing assays for therapeutic genome editing INDs
Funded at St. Jude Children's Research Hospital · NIH-11079534 · Data updated 2026-11-12
This study is working on making gene editing tools safer and more effective, which could help treat genetic diseases like sickle cell disease, so patients can have better treatment options in the future.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | May 17, 2023 |
| Project end | April 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $479,100 |
| NIH project number | 5U01AI176471-03 |
| Project ID | NIH-11079534 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients with genetic disorders such as sickle cell disease who may benefit from gene editing therapies.
✕Less likely to help
Patients with conditions that are not genetic in nature may not receive benefit from this research.
What this research studies
This research focuses on improving genome editing technologies that can potentially treat genetic diseases by modifying the DNA of living cells. The team aims to optimize and qualify new biochemical assays that ensure the safety and effectiveness of these gene therapies, particularly for investigational new drug submissions. By enhancing methods like CHANGE-seq and gRNA sequencing, the project seeks to provide reliable assessments of genome editing tools, ensuring they meet regulatory standards. Patients may benefit from these advancements as they could lead to safer and more effective treatments for conditions like sickle cell disease.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective gene therapies for genetic diseases.
How similar studies have gone: Other research has shown promise in optimizing gene editing technologies, but this specific approach is novel and aims to fill critical gaps in safety assessment.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Tsai, Shengdar — St. Jude Children's Research Hospital
- Study coordinator: Tsai, Shengdar
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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.