Understanding how specific gene mutations affect blood cell development and disorders.
Official title: Elucidating the Interplay Between DDX41 and CUX1 Mutations in Hematopoiesis in Danio Rerio and iPSCs
Funded at Albert Einstein College of Medicine · NIH-11071361 · Data updated 2026-11-12
This study is looking at how changes in certain genes might cause blood disorders like myelodysplastic syndromes and acute myeloid leukemia, using zebrafish and special human cells to understand how these changes affect blood cell development, which could help improve treatments for patients in the future.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Albert Einstein College of Medicine NIH-funded |
| Lab location | 1 site (Bronx, United States) |
| Project start | December 1, 2024 |
| Project end | March 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $53,974 |
| NIH project number | 1F31HL178296-01 |
| Project ID | NIH-11071361 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals with known DDX41 or CUX1 mutations, particularly those at risk for developing MDS or AML.
✕Less likely to help
Patients without these specific genetic mutations or those with unrelated blood disorders may not benefit from this research.
What this research studies
This research investigates the role of mutations in the DDX41 and CUX1 genes in the development of blood disorders, particularly myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). By using zebrafish and human induced pluripotent stem cells (iPSCs), the study aims to explore how these mutations interact and contribute to the dysfunction of hematopoietic stem and progenitor cells. Patients may benefit from insights gained about the genetic factors that lead to these conditions, potentially guiding future treatments or preventive strategies.
Why it matters
Potential benefit: If successful, this research could lead to improved understanding and management of blood disorders like MDS and AML.
How similar studies have gone: Previous research has shown that understanding genetic mutations can lead to significant advancements in treating hematologic malignancies, suggesting this approach has potential for success.
Where this research is happening
-
Albert Einstein College of Medicine
Bronx, United States Active
Researchers
- Principal investigator: Awad, Valerie — Albert Einstein College of Medicine
- Study coordinator: Awad, Valerie
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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.