ASO Therapy Targeting TILAM to Halt Liver Fibrosis
Official title: Long noncoding RNAs regulating liver fibrosis
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11364945 · Data updated 2026-11-12
Aims to develop antisense oligonucleotides that reduce a fibrosis-promoting RNA in liver cells to slow or stop scarring in chronic liver disease.
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 | August 17, 2019 |
| Project end | July 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $714,944 |
| NIH project number | 2R01DK116999-06 |
| Project ID | NIH-11364945 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are adults with chronic liver disease at risk of fibrosis or progression, who could participate in future trials testing TILAM-targeting ASOs.
✕Less likely to help
Patients without active fibrosis or who are not candidates for ASO-based therapies may not benefit from this research.
What this research studies
This project focuses on stopping liver scarring by targeting a specific RNA, TILAM, inside hepatic stellate cells. Researchers will design and test antisense therapies that deplete TILAM in these cells to prevent activation and excessive scar tissue formation. The work combines cellular models and molecular tools to identify the best ASO sequences and chemical tweaks for effective delivery to the liver. If successful, these ASOs could become a new treatment approach to protect liver function in people with chronic liver disease.
Why it matters
Potential benefit: If successful, this approach could slow or prevent liver fibrosis and preserve liver health in patients with chronic liver disease.
How similar studies have gone: Prior work shows that targeting disease-associated RNAs with ASOs can modulate fibrotic processes in preclinical models, supporting potential translational benefit.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Mullen, Alan C — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Mullen, Alan C
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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.