Creating advanced tools for precise gene editing
Engineering Efficient and Controllable Base Editors
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-10830957
This study is working on new tools to help scientists edit genes more accurately, especially in immune cells called B-cells, which could lead to better treatments for diseases like cancer while making sure they don’t cause any unexpected changes in the DNA.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-10830957 on ClinicalTrials.gov |
What this research studies
This research focuses on developing innovative DNA deaminase enzymes that can be precisely controlled for gene editing purposes. By enhancing the activity of these enzymes, the project aims to improve the accuracy and efficiency of editing specific genes, particularly in B-cells, which are crucial for immune responses. The approach involves using modified CRISPR/Cas proteins to direct these enzymes to specific genomic locations, potentially allowing for targeted treatments of various diseases, including cancers. The research also addresses safety concerns related to the use of these enzymes, ensuring that they do not cause unintended mutations.
Who could benefit from this research
Good fit: Ideal candidates for this research are patients with genetic mutations or cancers that could benefit from targeted gene editing therapies.
Not a fit: Patients with conditions that do not involve genetic mutations or those who are not candidates for gene therapy may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and safer gene therapies for patients with genetic disorders or cancers.
How similar studies have performed: Other research has shown promise in using gene editing technologies like CRISPR, indicating that this approach could lead to significant advancements in the field.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: KOHLI, RAHUL MANU — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: KOHLI, RAHUL MANU
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.
Conditions: Cancers, neoplasm/cancer