Study uncovers potential therapeutic targets for low platelet blood disorders
MD Anderson Research Highlight July 01, 2025
Thrombocytopenia is a blood disorder characterized by low levels of blood platelets. These tiny blood cells, which help with clotting, are made in the bone marrow and spleen by cells called megakaryocytes. However, little is known about the regulatory factors involved in platelet formation, which could be used to develop more effective therapeutic strategies for thrombocytopenia. MLL3 and MLL4 are part of a protein complex that modifies histones involved in DNA packaging to control gene activity. Both genes are frequently mutated in various cancer types, leading Margarida Santos, Ph.D., and colleagues to examine their role in platelet formation. The researchers generated models missing the MLL3 and MLL4 genes. They found models missing only one gene had normal platelet counts, but those missing both had impaired megakaryocyte maturation and developed severe macrothrombocytopenia, with fewer and larger-than-normal platelets that easily self-destruct. These results provide epigenetic insights into blood disorders involving low platelets, highlighting MLL3 and MLL4 as potential therapeutic targets. Learn more in Nature Communications.
These findings provide novel targets that will aid in the future expansion of platelets and therapies aimed at enhancing platelet production. This is crucial for our patients with cancer, as platelets are always in a great demand in a cancer setting.