Using microRNA to help hearts affected by dilated cardiomyopathy
Official title: MicroRNA Control of Dilated Cardiomyopathy
Funded at Stanford University · NIH-11235201 · Data updated 2026-11-12
They are trying small RNA molecules to help heart muscle cells work better in people with inherited dilated cardiomyopathy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | December 12, 2023 |
| Project end | November 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $591,214 |
| NIH project number | 5R01HL130840-07 |
| Project ID | NIH-11235201 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with inherited dilated cardiomyopathy caused by known gene variants (for example TNNT2 mutations) would be the most relevant candidates for future trials based on this work.
✕Less likely to help
People whose heart failure is due to non-genetic causes or who have very advanced disease may not benefit from these approaches.
What this research studies
Researchers are using patient-derived stem cells turned into heart muscle cells to find synthetic microRNAs that restore normal contraction in cells with genetic DCM. In earlier work they found two synthetic microRNAs that returned contractility to patient cells to levels similar to fixing the underlying mutation with CRISPR. The team biochemically mapped the microRNA targets and found about 203 candidate genes, with inhibition of many of those genes restoring function in mutated cells from different donors. The current project aims to figure out how those genes cause dysfunction and to validate the most promising targets as potential ways to treat inherited DCM.
Why it matters
Potential benefit: If successful, this could point to new therapies that restore heart muscle function for people with genetic dilated cardiomyopathy.
How similar studies have gone: Previous lab studies showed synthetic microRNAs could normalize contraction in patient-derived heart cells similar to CRISPR correction, but this is still early-stage and has not been tested in people.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Mercola, Mark — Stanford University
- Study coordinator: Mercola, Mark
Related research
Other NIH-funded projects on the same topics.
-
Discovery of rare genetic variants linked to dilated cardiomyopathy using big biobank data
A large-scale genetic study will look for rare gene variants that affect dilated cardiomyopathy to improve how we diagnose and manage the condition.
-
Understanding how muscle ribosome mutations cause severe neonatal dilated cardiomyopathy and ways to treat it
This grant focuses on a rare genetic heart condition in newborns caused by mutations in a muscle-specific ribosomal protein, with hopes to find new ways to diagnose and treat it.
-
Unlocking how rare genetic changes cause dilated cardiomyopathy
A large study will look at genetic data from many people to understand which rare variants affect dilated cardiomyopathy and how this changes risk and treatment.
-
Role of Lmod2 in heart muscle and dilated cardiomyopathy
This NIH grant explores how a heart-specific protein (Lmod2) helps heart cells contract properly and how its problems can cause dilated cardiomyopathy in infants and children.
-
Targeting DNA Damage Pathways in LMNA-Related Heart Disease
A study to understand how damaged DNA and inflammatory signals contribute to dilated cardiomyopathy caused by LMNA mutations and explore therapies that can protect the heart.
-
Using miRNA to find new treatment paths for dilated cardiomyopathy
Researchers are studying heart-muscle disease (dilated cardiomyopathy) to discover miRNA-based clues that could lead to targeted therapies for patients.
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.