Modifying gene expression using human-derived RNA technologies
Official title: Engineering human-derived programmable RNA effectors to retune gene expression
Funded at University of Chicago · NIH-11097179 · Data updated 2026-11-12
This study is exploring a new way to safely turn genes on and off to help treat diseases like cancer and nerve disorders, starting with a gene linked to certain nerve problems, and it's designed to be gentle on your immune system.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Chicago NIH-funded |
| Lab location | 1 site (Chicago, United States) |
| Project start | June 1, 2023 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $392,065 |
| NIH project number | 5R01EB035016-03 |
| Project ID | NIH-11097179 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with conditions related to gene dosage imbalances, such as certain cancers, cardiovascular diseases, and neuropathies.
✕Less likely to help
Patients with diseases not linked to gene expression imbalances may not benefit from this research.
What this research studies
This research focuses on developing a new technology to adjust gene expression in a precise and reversible manner, which is crucial for treating diseases linked to gene imbalances, such as cancers and neurological disorders. The approach utilizes a novel RNA-targeting system inspired by CRISPR, designed to minimize immune reactions by using human proteins. By engineering these RNA effectors, the research aims to create effective treatments that can either activate or deactivate specific genes associated with various diseases. The initial target for testing this technology will be the PMP22 gene, which is known to have significant implications in certain neuropathies.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that effectively manage diseases caused by gene expression imbalances.
How similar studies have gone: Other research has shown promise with similar RNA-targeting technologies, indicating potential for success in this novel approach.
Where this research is happening
-
University of Chicago
Chicago, United States Active
Researchers
- Principal investigator: Dickinson, Bryan — University of Chicago
- Study coordinator: Dickinson, Bryan
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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.