Developing mRNA therapies for a deadly pediatric brain tumor
Official title: Enabling mRNA Therapies for Pediatric High-Grade Gliomas
Funded at Mayo Clinic Rochester · NIH-10947149 · Data updated 2026-11-12
This study is exploring a new way to treat a tough brain tumor called diffuse midline glioma in kids by using special particles to deliver gene therapy directly to the tumor, aiming to improve how we fight this disease.
Quick facts
| Grant type | Career grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | September 18, 2024 |
| Project end | August 31, 2026 |
| Funding year | 2024 |
| Award (this year) | $142,452 |
| NIH project number | 1K99NS138490-01 |
| Project ID | NIH-10947149 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with diffuse midline glioma.
✕Less likely to help
Patients with other types of brain tumors or those over the age of 11 may not benefit from this research.
What this research studies
This research focuses on diffuse midline glioma (DMG), a highly aggressive brain tumor in children that currently has no effective treatment. The study aims to utilize mRNA-based gene therapies to target a specific mutation (H3K27M) that drives the disease. Researchers will develop lipid nanoparticles to deliver mRNA directly to the tumor site, overcoming significant barriers in the brain. The project will involve testing various delivery methods and understanding how these nanoparticles interact with brain cells to improve treatment efficacy.
Why it matters
Potential benefit: If successful, this research could lead to a novel and effective treatment for pediatric patients suffering from DMG.
How similar studies have gone: Previous research has shown promise in using mRNA therapies for other cancers, but this specific approach for DMG is novel and untested.
Where this research is happening
-
Mayo Clinic Rochester
Rochester, United States Active
Researchers
- Principal investigator: Yerneni, Saigopalakrishna — Mayo Clinic Rochester
- Study coordinator: Yerneni, Saigopalakrishna
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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.