Controlling gene therapies using synthetic RNA switches
Official title: Synthetic RNA Switch-Based Temporal and Dose Control of in Vivo Gene Therapies
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11104441 · Data updated 2026-11-12
This study is working on a new way to make gene therapies safer and more effective by using special switches that can turn the therapy on or off, helping to adjust treatment based on how your condition changes.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project start | March 1, 2025 |
| Project end | February 28, 2029 |
| Funding year | 2025 |
| Award (this year) | $540,524 |
| NIH project number | 1R01EB037025-01 |
| Project ID | NIH-11104441 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research would include individuals with conditions that could benefit from gene therapy, particularly those requiring long-term treatment adjustments.
✕Less likely to help
Patients who do not require gene therapy or those with conditions that are not amenable to this type of treatment may not benefit from this research.
What this research studies
This research focuses on developing a new method to control gene therapies that use adeno-associated viral (AAV) vectors. By creating synthetic RNA switches, the team aims to regulate when and how much therapeutic gene expression occurs in the body, allowing for adjustments based on disease progression or side effects. The approach involves using a small ribozyme that can be inserted into the gene therapy vector, which can be activated or deactivated by a safe morpholino oligonucleotide. This could lead to more effective and safer long-term treatments for various diseases.
Why it matters
Potential benefit: If successful, this research could provide patients with more personalized and adaptable gene therapies that can be fine-tuned over time.
How similar studies have gone: While some research has explored gene therapy regulation, this approach using synthetic RNA switches is relatively novel and has not been widely tested.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Zhong, Guocai — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Zhong, Guocai
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.