Understanding how gene changes cause childhood brain cancer
Developmental transcription factors in Drosha-driven cancer
This research aims to understand how specific gene mutations lead to pineoblastoma, a type of childhood brain cancer, to find new ways to treat it.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ut Southwestern Medical Center NIH-funded |
| Lab location | 1 site (Dallas, United States) |
| Project ID | NIH-11176811 on NIH RePORTER |
What this research studies
Pineoblastoma is a childhood brain cancer caused by changes in genes called DROSHA and DICER1, but we don't fully understand how these changes lead to cancer or how to treat them. Our team has created a special mouse model that develops tumors similar to human pineoblastoma to learn more. We've found that certain developmental genes are overactive in these tumors, and we want to see if one of these genes, called Onecut2, is essential for the cancer to grow. By studying how Onecut2 works with other genes and testing its role in patient-derived tumor models, we hope to uncover new treatment possibilities.
Who could benefit from this research
Good fit: This foundational research could eventually benefit children diagnosed with pineoblastoma, especially those with DROSHA or DICER1 gene mutations.
Not a fit: Patients with other types of brain cancer not linked to DROSHA or DICER1 mutations may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could identify new targets for therapies that specifically treat pineoblastoma caused by DROSHA or DICER1 mutations.
How similar studies have performed: The specific pathways by which these mutations cause cancer are not fully understood, and there are currently no targeted therapies for these mutations, making this a novel approach.
Where this research is happening
Dallas, United States
- Ut Southwestern Medical Center — Dallas, United States (Active)
Researchers
- Principal investigator: Chen, Kenneth Sung-Man — Ut Southwestern Medical Center
- Study coordinator: Chen, Kenneth Sung-Man
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.