Developing advanced tools for precise gene editing using CRISPR technology
Programmable gene integration and cell engineering with CRISPR-directed integrases
['FUNDING_R01'] · BRIGHAM AND WOMEN'S HOSPITAL · NIH-11097018
This study is working on new ways to edit genes more effectively in different types of cells, which could help improve treatments for various health conditions by allowing for precise changes to DNA without depending on the cell's natural repair processes.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BRIGHAM AND WOMEN'S HOSPITAL (nih funded) |
| Locations | 1 site (BOSTON, UNITED STATES) |
| Trial ID | NIH-11097018 on ClinicalTrials.gov |
What this research studies
This research focuses on creating new tools for gene editing that can work efficiently across various cell types. It aims to improve upon existing CRISPR methods by developing integrase-based systems that allow for the precise insertion of large genes without relying on the cell's DNA repair mechanisms. By combining computational methods with biochemical techniques, the project seeks to discover and engineer new proteins that can facilitate these gene edits. This could lead to significant advancements in both basic biological research and therapeutic applications, such as engineered cell therapies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic conditions that could benefit from advanced 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 provide patients with more effective gene therapies that can treat a wider range of genetic disorders.
How similar studies have performed: Other research has shown promising results with CRISPR technology, but this approach of using integrase-based systems is relatively novel and untested.
Where this research is happening
BOSTON, UNITED STATES
- BRIGHAM AND WOMEN'S HOSPITAL — BOSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ABUDAYYEH, OMAR O — BRIGHAM AND WOMEN'S HOSPITAL
- Study coordinator: ABUDAYYEH, OMAR O
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.