Investigating the safety and effectiveness of CRISPR gene editing techniques
Official title: Replication Study of Off-Target Assays for Guide RNAs in CRISPR-Based Therapeutics
Funded at University of Wisconsin-Madison · NIH-11200059 · Data updated 2026-11-12
This study is looking at ways to make CRISPR gene editing safer and more effective for people with conditions like Leber's congenital amaurosis by checking for any unintended effects that might happen during the process.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project start | December 1, 2024 |
| Project end | April 30, 2025 |
| Funding year | 2025 |
| Award (this year) | $77,750 |
| NIH project number | 3U19NS132296-02S2 |
| Project ID | NIH-11200059 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic conditions, particularly those affected by Leber's congenital amaurosis.
✕Less likely to help
Patients with conditions unrelated to genetic disorders or those not amenable to CRISPR-based therapies may not benefit from this research.
What this research studies
This research focuses on improving CRISPR-based gene editing by examining the off-target effects that can occur when using guide RNAs. By comparing results from different laboratories, the study aims to identify the most reliable assays for assessing these off-target effects. This will involve using contract research organizations to conduct genome-wide assays, which will help establish best practices for the safe application of CRISPR therapies. The ultimate goal is to enhance the safety and efficacy of gene editing treatments for conditions like Leber's congenital amaurosis.
Why it matters
Potential benefit: If successful, this research could lead to safer and more effective CRISPR therapies for patients with genetic disorders.
How similar studies have gone: Previous research has shown promise in improving CRISPR techniques, but this specific approach to standardizing off-target assays is relatively novel.
Where this research is happening
-
University of Wisconsin-Madison
Madison, United States Active
Researchers
- Principal investigator: Saha, Krishanu — University of Wisconsin-Madison
- Study coordinator: Saha, Krishanu
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.