Using precise genetic tools to correct single point mutations in diseases.
Precision Deaminases to Expand the Scope of Diseases Amenable to CRISPR-mediated Correction
['FUNDING_FELLOWSHIP'] · UNIVERSITY OF CALIFORNIA BERKELEY · NIH-11037911
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
| Phase | ['FUNDING_FELLOWSHIP'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF CALIFORNIA BERKELEY (nih funded) |
| Locations | 1 site (BERKELEY, UNITED STATES) |
| Trial ID | NIH-11037911 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders caused by specific point mutations, such as severe combined immunodeficiency (SCID).
Not a fit: Patients with genetic disorders that do not involve single point mutations or those with complex genetic conditions may not benefit from this research.
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 performed: 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
BERKELEY, UNITED STATES
- UNIVERSITY OF CALIFORNIA BERKELEY — BERKELEY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: DOHERTY, ERIN E — UNIVERSITY OF CALIFORNIA BERKELEY
- Study coordinator: DOHERTY, ERIN E
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.