Investigating the origins and treatment options for aggressive pediatric brain tumors.
Generating hiPSC-derived ATRT models to investigate cell of origin and identify therapeutic vulnerabilities
This study is looking at a tough type of brain cancer in kids called atypical teratoid rhabdoid tumors (ATRTs) to learn more about how they grow and what makes them tick, with the hope of finding better treatments to help children who are affected.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Diego NIH-funded |
| Lab location | 1 site (La Jolla, United States) |
| Project ID | NIH-10996034 on NIH RePORTER |
What this research studies
This research focuses on atypical teratoid rhabdoid tumors (ATRTs), which are aggressive brain cancers in children that currently lack effective treatment options. The study aims to understand the biology of these tumors by generating models from human induced pluripotent stem cells (hiPSCs) to identify their cell of origin and potential therapeutic vulnerabilities. By examining how these tumors develop and their unique characteristics, the research seeks to uncover new strategies for treatment that could improve outcomes for affected children. The approach includes advanced techniques like CRISPR to manipulate genes and study their effects on tumor behavior.
Who could benefit from this research
Good fit: Ideal candidates for this research are children under 11 years old diagnosed with atypical teratoid rhabdoid tumors.
Not a fit: Patients with other types of brain tumors or those over the age of 11 may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the development of targeted therapies that improve survival rates and reduce long-term side effects for children with ATRTs.
How similar studies have performed: While research on ATRTs is ongoing, this specific approach using hiPSC models and CRISPR technology is relatively novel and has not been extensively tested in this context.
Where this research is happening
La Jolla, United States
- University of California, San Diego — La Jolla, United States (Active)
Researchers
- Principal investigator: Wang, Clark Gold — University of California, San Diego
- Study coordinator: Wang, Clark Gold
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.