Enhanced Epigenome Editing Tools to Activate Gene Expression in Living Systems
Official title: Enhanced Epigenome Editing Technologies for Controlling Mammalian Gene Expression
Funded at University of Illinois at Urbana-Champaign · NIH-11387406 · Data updated 2026-11-12
Aims to build new AAV-friendly epigenome editors that turn on human gene expression inside living cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Urbana-Champaign NIH-funded |
| Lab location | 1 site (Champaign, United States) |
| Project start | May 14, 2026 |
| Project end | February 28, 2030 |
| Funding year | 2026 |
| Award (this year) | $526,483 |
| NIH project number | 1R01GM165051-01 |
| Project ID | NIH-11387406 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be individuals with conditions where targeted gene activation could be therapeutically beneficial, pending future clinical development.
✕Less likely to help
Patients not receiving a gene-activation therapy or those without a condition linked to targetable gene activation may not benefit.
What this research studies
This project develops advanced tools to turn on genes inside living organisms using a compact epigenome editing system compatible with AAV delivery. By combining protein engineering, machine learning, and improved viral vector design, the work seeks to identify p300 variants that reliably activate genes in mammalian cells. It also creates predictive models to choose the best target sites for activation and designs ultra-compact editors suitable for in vivo use. The ultimate goal is a practical toolkit for activating genes with precision in animal and potentially human contexts.
Why it matters
Potential benefit: If successful, patients could benefit from future gene-activation therapies for diseases where turning on beneficial genes is promising.
How similar studies have gone: Related work shows that refined epigenome editors can modulate gene expression, though challenges remain in efficiency and delivery.
Where this research is happening
-
University of Illinois at Urbana-Champaign
Champaign, United States Active
Researchers
- Principal investigator: Perez-Pinera, Pablo — University of Illinois at Urbana-Champaign
- Study coordinator: Perez-Pinera, Pablo
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.