RNA tools to measure and control ADAR activity
Nucleic acid modulators and theranostics for ADAR
Developing RNA-based tools to measure and change ADAR activity to help people with certain genetic clotting disorders and some cancers.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project ID | NIH-11166670 on NIH RePORTER |
What this research studies
We are creating RNA "reporters" that light up to map ADAR activity inside living animals so we can see where and when ADAR is active. We plan to boost or guide the body's own ADAR using engineered guide RNAs and immune-stimulating DNA pieces, and we will attach therapeutic RNA/DNA to albumin so it travels to the right tissues. We will test these delivery methods and theranostic reporters in mouse models of Factor V Leiden blood clotting disorder and metastatic melanoma. The goal is to learn how ADAR varies across tissues and people to design personalized RNA-based treatments and to see if changing ADAR can make cancer immunotherapy work better.
Who could benefit from this research
Good fit: People affected by Factor V Leiden thrombophilia or metastatic melanoma, or those interested in future ADAR-targeting therapies, would be the eventual candidates for clinical follow-up from this work.
Not a fit: Patients with conditions unrelated to ADAR biology or those seeking immediate clinical treatment are unlikely to benefit directly because the work is preclinical.
Why it matters
Potential benefit: If successful, this could enable noninvasive tests of ADAR activity and new personalized RNA therapies for diseases caused by certain genetic mutations and for improving cancer immunotherapy.
How similar studies have performed: Related preclinical studies of ADAR editing and ADAR inhibition have shown promise, but clinical benefits in people are not yet established.
Where this research is happening
Ann Arbor, United States
- University of Michigan at Ann Arbor — Ann Arbor, United States (Active)
Researchers
- Principal investigator: Zhu, Guizhi — University of Michigan at Ann Arbor
- Study coordinator: Zhu, Guizhi
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.