Understanding how specific gene mutations affect blood cell development and disorders.
Elucidating the Interplay Between DDX41 and CUX1 Mutations in Hematopoiesis in Danio Rerio and iPSCs
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 ID | NIH-11071361 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Ideal candidates for this research include individuals with known DDX41 or CUX1 mutations, particularly those at risk for developing MDS or AML.
Not a fit: Patients without these specific genetic mutations or those with unrelated blood disorders may not benefit from this research.
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 performed: 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
Bronx, United States
- Albert Einstein College of Medicine — Bronx, United States (Active)
Researchers
- Principal investigator: Awad, Valerie — Albert Einstein College of Medicine
- Study coordinator: Awad, Valerie
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.