Understanding how GATA2 affects blood cancers and MonoMAC syndrome

The role of GATA2 in myeloid malignancies and MonoMAC syndrome

NIH-funded research Indiana University Indianapolis · NIH-10783945

This study is looking at how changes in the GATA2 gene might influence the development of acute myeloid leukemia and MonoMAC syndrome, with the hope of finding new treatments for people affected by these conditions.

Quick facts

Grant typeNIH-funded research
Study typeNIH-funded research
Funding institutionIndiana University Indianapolis NIH-funded
Lab location1 site (Indianapolis, United States)
Project IDNIH-10783945 on NIH RePORTER

What this research studies

This research investigates the role of the GATA2 gene in the development of acute myeloid leukemia (AML) and MonoMAC syndrome. By studying mutations in GATA2, which can either suppress or promote cancer, the researchers aim to uncover how these genetic changes affect blood cell formation and function. The project utilizes advanced techniques like single-cell RNA sequencing to analyze gene expression and protein interactions in specially developed mouse models that mimic human conditions. The ultimate goal is to translate these findings into potential therapeutic strategies for patients.

Who could benefit from this research

Good fit: Ideal candidates for this research include individuals diagnosed with acute myeloid leukemia or those with MonoMAC syndrome, particularly those with known GATA2 mutations.

Not a fit: Patients without acute myeloid leukemia or MonoMAC syndrome, or those without GATA2 mutations, may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to new treatments for patients with acute myeloid leukemia and MonoMAC syndrome.

How similar studies have performed: Previous research has shown promising results in understanding the role of genetic mutations in blood cancers, suggesting that this approach could yield valuable insights.

Where this research is happening

Indianapolis, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.