Using microRNA to help hearts affected by dilated cardiomyopathy
MicroRNA Control of Dilated Cardiomyopathy
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 ID | NIH-11235201 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: People whose heart failure is due to non-genetic causes or who have very advanced disease may not benefit from these approaches.
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 performed: 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, United States
- Stanford University — Stanford, United States (Active)
Researchers
- Principal investigator: Mercola, Mark — Stanford University
- Study coordinator: Mercola, Mark
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.