How the MN1 protein drives aggressive acute myeloid leukemia
IDP mediated transcriptional stabilization as a cause of AML
This work looks at how a protein called MN1 helps cause aggressive acute myeloid leukemia, especially in patients whose disease resists standard treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-11303396 on NIH RePORTER |
What this research studies
As a patient, I would be told that researchers are studying MN1, an unusually flexible protein that is often overactive in AML and linked to worse outcomes. They are combining mouse models where MN1 causes leukemia with lab experiments that map where MN1 and the BAF chromatin complex bind DNA and how that keeps stem-like gene programs turned on. The team uses biochemical tests, chromatin and gene-activity profiling, and cell experiments to see how MN1 creates high local concentrations of transcription machinery that may be targetable. Their approach aims to find molecular steps that could be blocked by new therapies for MN1-driven AML.
Who could benefit from this research
Good fit: People with acute myeloid leukemia, particularly those whose leukemia shows high MN1 expression or who have relapsed or refractory disease, would be the most relevant candidates.
Not a fit: Patients without AML or with AML driven by unrelated molecular causes are unlikely to gain direct benefit from this specific research.
Why it matters
Potential benefit: If successful, the work could identify new targets for drugs to treat patients with MN1-high, treatment-resistant AML.
How similar studies have performed: Previous studies have linked MN1 to poor outcomes and produced MN1-driven leukemia in mice, but directly targeting MN1's disordered interactions is a novel and unproven approach.
Where this research is happening
Philadelphia, United States
- Children's Hosp of Philadelphia — Philadelphia, United States (Active)
Researchers
- Principal investigator: Bernt, Kathrin M — Children's Hosp of Philadelphia
- Study coordinator: Bernt, Kathrin 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.