How ASXL1 mutations change aging blood stem cells

Molecular and Cellular Mechanisms of Mutant ASXL1-driven Clonal Hematopoiesis

NIH-funded research University of Wisconsin-Madison · NIH-11180108

Researchers are looking at how a common ASXL1 gene mutation causes age-related changes in blood stem cells and whether targeting cell-survival pathways can reduce harmful cell expansion.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionUniversity of Wisconsin-Madison NIH-funded
Lab location1 site (Madison, United States)
Project IDNIH-11180108 on NIH RePORTER

What this research studies

From a patient's view, this work studies a common age-related condition called clonal hematopoiesis (CHIP) driven by ASXL1 mutations that can raise the risk of blood cancers. The team uses mouse models carrying the ASXL1 hotspot mutation and compares young versus old bone marrow environments to see how inflammation, senescent stromal and endothelial cells, and epigenetic changes help mutant stem cells expand. They test whether blocking anti-apoptotic proteins (for example with the drug ABT-263) can reduce survival of senescent niche cells and alter mutant cell growth. Findings could point to ways to interrupt the unhealthy bone marrow environment that helps pre-leukemic clones grow.

Who could benefit from this research

Good fit: People most relevant to this research are older adults with clonal hematopoiesis, especially those known to carry ASXL1 mutations or unexplained age-related changes in blood counts.

Not a fit: Young healthy people without CHIP or patients whose blood conditions are unrelated to ASXL1 mutations are unlikely to get direct benefit from this specific work.

Why it matters

Potential benefit: If successful, this work could lead to new ways to prevent or slow progression of ASXL1-driven clonal hematopoiesis and reduce future blood cancer risk by targeting survival pathways in the bone marrow niche.

How similar studies have performed: Related lab and animal studies have shown that BCL-2/BCL-XL inhibitors can clear senescent cells, but applying these approaches specifically to ASXL1-driven CHIP is a newer direction.

Where this research is happening

Madison, 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.