Targeting childhood cancer vulnerabilities with CRISPR-based screens
Official title: Targeting Pediatric Cancer Vulnerabilities
Funded at Dana-Farber Cancer Inst · NIH-11399729 · Data updated 2026-11-12
A pediatric cancer research project uses CRISPR screens to find new, druggable weaknesses in childhood leukemias to guide safer, targeted therapies.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 19, 2023 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $1,066,178 |
| NIH project number | 5R35CA283977-04 |
| Project ID | NIH-11399729 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children and young adults with or at risk for pediatric AML or related transcription factor-driven leukemias who may benefit from future targeted therapies or participation in related studies.
✕Less likely to help
Patients with cancers not driven by the explored pediatric targets or without access to emerging targeted therapies may not benefit directly.
What this research studies
This project aims to uncover key transcription factor-related vulnerabilities in pediatric cancers, focusing on fusion-driven acute myeloid leukemia. Researchers will use CRISPR-based screening to validate targets and identify initial chemical leads for precision therapies in children. The work is led by a pediatric oncologist-innovator at Dana-Farber, with a clear aim to de-risk high-priority pediatric targets and move beyond traditional chemotherapy and radiation. Findings could inform future treatment options that are more effective and have fewer long-term side effects for young patients.
Why it matters
Potential benefit: If successful, the research could reveal new targets and early drug leads that enable safer, more effective treatments for children with acute myeloid leukemia.
How similar studies have gone: Previous studies using CRISPR screens and target validation in cancer have identified actionable targets, supporting the approach, though pediatric-specific successes remain an active area.
Where this research is happening
-
Dana-Farber Cancer Inst
Boston, United States Active
Researchers
- Principal investigator: Stegmaier, Kimberly — Dana-Farber Cancer Inst
- Study coordinator: Stegmaier, Kimberly
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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.