Investigating the role of a specific gene in myelofibrosis
Official title: The Role of PTPN11 in Myelofibrosis
Funded at University of Virginia · NIH-10545076 · Data updated 2026-11-12
This study is looking at how a gene called PTPN11 might help us find new treatments for myelofibrosis, a serious blood disorder, by seeing how blocking this gene affects blood cell growth in patients, especially those with a common mutation.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Virginia NIH-funded |
| Lab location | 1 site (Charlottesville, United States) |
| Project start | January 1, 2020 |
| Project end | June 30, 2025 |
| Funding year | 2023 |
| Award (this year) | $399,195 |
| NIH project number | 5R01HL149893-04 |
| Project ID | NIH-10545076 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with myelofibrosis, particularly those with the JAK2V617F mutation.
✕Less likely to help
Patients with myelofibrosis who do not have the JAK2V617F mutation or other related mutations may not benefit from this research.
What this research studies
This research focuses on myelofibrosis, a severe blood disorder characterized by the buildup of fibrous tissue in the bone marrow. The study aims to explore the role of the PTPN11 gene, which may be a new target for treatment. Researchers will investigate how inhibiting PTPN11 affects the growth of blood cells in patients with myelofibrosis, particularly those with a common mutation known as JAK2V617F. By using advanced genetic techniques, the team hopes to identify new therapeutic strategies that could improve patient outcomes.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that effectively target myelofibrosis and improve survival rates for patients.
How similar studies have gone: Previous research has shown promise in targeting similar pathways in other blood disorders, suggesting potential for success in this approach.
Where this research is happening
-
University of Virginia
Charlottesville, United States Active
Researchers
- Principal investigator: Mohi, Golam — University of Virginia
- Study coordinator: Mohi, Golam
Related research
Other NIH-funded projects on the same topics.
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Strategies to deplete myelofibrosis stem cells using HDM2/p53 pathway
A patient-focused program tests a therapy that may selectively reduce disease-causing blood stem cells in people with myelofibrosis.
-
Megakaryocyte changes in myelofibrosis and its progression to leukemia
A scientist-guided project will study how abnormal megakaryocytes and signals like TGF-β and IL-13 may drive progression from myelofibrosis toward acute leukemia, with potential implications for future treatments.
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Molecular clues and new therapies for myelofibrosis and related blood cancers
This grant supports research to understand what drives myelofibrosis and related disorders and to develop new treatment ideas for patients.
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Myelofibrosis and the bone marrow environment: how inflammation changes blood production
A doctor-researcher is studying how inflammation in the bone marrow affects the blood-forming system in people with myeloproliferative neoplasms (MPNs) to find ways to prevent scarring and leukemia.
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Early detection of treatment response in myelofibrosis using a new imaging technology
A new, quick imaging method could tell patients with myelofibrosis early whether a treatment is working.
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MYC-driven vulnerability in myelofibrosis could guide new therapies
A new project is exploring how the MYC protein contributes to myelofibrosis and whether it can be targeted to help people with this blood cancer.
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.