Using precise genetic tools to correct single point mutations in diseases.
Official title: Precision Deaminases to Expand the Scope of Diseases Amenable to CRISPR-mediated Correction
Funded at University of California Berkeley · NIH-11037911 · Data updated 2026-11-12
This study is exploring a new way to fix genetic mistakes that lead to disorders, using a special technique that makes precise changes to DNA, which could help improve treatments for conditions like immune system issues and brain diseases.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Berkeley NIH-funded |
| Lab location | 1 site (Berkeley, United States) |
| Project start | March 1, 2024 |
| Project end | February 28, 2027 |
| Funding year | 2025 |
| Award (this year) | $74,284 |
| NIH project number | 5F32GM153031-02 |
| Project ID | NIH-11037911 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals with genetic disorders caused by specific point mutations, such as severe combined immunodeficiency (SCID).
✕Less likely to help
Patients with genetic disorders that do not involve single point mutations or those with complex genetic conditions may not benefit from this research.
What this research studies
This research focuses on developing advanced genetic editing techniques to correct specific mutations that cause various genetic disorders. By utilizing a method called base editing, which employs a deaminase enzyme, the researchers aim to make precise changes to DNA without causing double-stranded breaks. This approach is particularly beneficial for stem cells, which are crucial for therapies targeting conditions like immunodeficiency and neurodegenerative diseases. The goal is to expand the range of treatable genetic disorders by improving the accuracy and efficiency of genetic corrections.
Why it matters
Potential benefit: If successful, this research could lead to new therapies for a wide range of genetic disorders that are currently untreatable.
How similar studies have gone: Other research has shown promising results with similar base editing techniques, indicating a strong potential for success in this area.
Where this research is happening
-
University of California Berkeley
Berkeley, United States Active
Researchers
- Principal investigator: Doherty, Erin E — University of California Berkeley
- Study coordinator: Doherty, Erin E
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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.