A new RNA drug to reduce heart scarring in scleroderma patients
Official title: Therapeutic preclinical development of novel RNA drug for scleroderma heart disease
Funded at Cedars-Sinai Medical Center · NIH-11339407 · Data updated 2026-11-12
Aims to test a small RNA therapy that could reduce heart fibrosis and improve heart function in people with scleroderma-related heart disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cedars-Sinai Medical Center NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project start | July 8, 2026 |
| Project end | April 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $581,138 |
| NIH project number | 1R01HL185055-01 |
| Project ID | NIH-11339407 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with systemic sclerosis (scleroderma) who have or are at risk for heart involvement.
✕Less likely to help
Patients without scleroderma heart involvement may not benefit from this RNA therapy.
What this research studies
This project looks at TY1, a tiny synthetic RNA that can be given in a protective oral formulation to reach the heart. The researchers will study how TY1 affects heart tissue in mouse models of scleroderma and in human cells from people with the condition. They will focus on how TY1 may lessen fibrosis and protect the heart by influencing immune and DNA-damage pathways. If TY1 works in these models, it could move toward helping patients with scleroderma-caused heart disease.
Why it matters
Potential benefit: If successful, TY1 could become a treatment to reduce heart scarring and improve heart function in scleroderma patients.
How similar studies have gone: Preclinical studies have shown antifibrotic effects in mouse models and human cells, with a novel mechanism involving DNA damage response.
Where this research is happening
-
Cedars-Sinai Medical Center
Los Angeles, United States Active
Researchers
- Principal investigator: Ciullo, Alessandra — Cedars-Sinai Medical Center
- Study coordinator: Ciullo, Alessandra
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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.