Childhood acute myeloid leukemia linked to UBTF gene duplications
UBTF Tandem Duplications in Pediatric Acute Myeloid Leukemia
This project looks at a specific UBTF gene change in children with acute myeloid leukemia to understand why their disease returns and resists treatment.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project ID | NIH-11162534 on NIH RePORTER |
What this research studies
This project focuses on a gene change called a UBTF exon 13 tandem duplication found in a subset of children with AML. Researchers will analyze tumor samples and clinical records from children with newly diagnosed and relapsed AML to determine how often UBTF-TD occurs and how it relates to outcomes. In the lab they will study patient-derived cells and molecular interactions to see how UBTF-TD changes blood cell growth and connects to other leukemia-related proteins. The team plans to link these biological findings to clinical risk so this group of patients can be better classified and considered for targeted approaches.
Who could benefit from this research
Good fit: Children with acute myeloid leukemia, particularly those with relapsed disease or whose tumor testing shows UBTF exon 13 tandem duplication or associated mutations like FLT3-ITD or WT1, are the most relevant candidates.
Not a fit: Adults without UBTF-TD and patients whose leukemia lacks UBTF-TD are unlikely to get direct benefit from this specific project.
Why it matters
Potential benefit: If successful, this work could help identify children at higher risk of relapse and point to new, more targeted treatment options.
How similar studies have performed: The investigators previously discovered UBTF-TD in pediatric AML and showed it can drive leukemia in laboratory models, but translating this into clinical therapies is still early and novel.
Where this research is happening
Memphis, United States
- St. Jude Children's Research Hospital — Memphis, United States (Active)
Researchers
- Principal investigator: Klco, Jeffery M — St. Jude Children's Research Hospital
- Study coordinator: Klco, Jeffery M
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.