Improving RNA medicines to treat genetic diseases in more body parts
Chemical engineering of therapeutic RNAs for extrahepatic delivery
This work aims to make RNA-based medicines, which are designed to treat genetic conditions, work effectively in many different body tissues beyond the liver.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project ID | NIH-11011916 on NIH RePORTER |
What this research studies
Our bodies are made of many different tissues, and while some RNA medicines work well in the liver, we want them to help other parts of the body too. This project focuses on changing the chemical structure of these RNA medicines to make them more powerful and long-lasting in various tissues. We are also working on ways to guide these medicines specifically to the affected areas. By improving how these medicines are delivered, we hope to open up new treatment options for a wider range of genetic diseases.
Who could benefit from this research
Good fit: Patients with genetically defined disorders affecting tissues beyond the liver could potentially benefit from future therapies developed through this research.
Not a fit: Patients whose conditions are not genetically defined or do not involve the specific RNA pathways targeted by these medicines may not receive direct benefit.
Why it matters
Potential benefit: If successful, this work could lead to new RNA-based treatments for genetic disorders that affect tissues outside the liver, offering hope for conditions currently without effective therapies.
How similar studies have performed: Similar RNA-based approaches have already shown success in treating liver-related conditions, and some are currently in clinical trials for other tissues, indicating a promising foundation for this work.
Where this research is happening
Worcester, United States
- Univ of Massachusetts Med Sch Worcester — Worcester, United States (Active)
Researchers
- Principal investigator: Khvorova, Anastasia — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Khvorova, Anastasia
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.