The evolution of epigenetic therapy in myelodysplastic syndromes and acute myeloid leukemia

Journal Name
Seminars in Hematology
Primary Author
Gonzalez-Lugo J
Author(s)
Jesus D Gonzalez-Lugo, Samarpana Chakraborty, Amit Verma, Aditi Shastri
Original Publication Date

Mutations in the group of epigenetic modifiers are the largest group of mutated genes in Myelodysplastic Syndromes: (my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at… (MDS) and are very frequently found in Acute Myeloid Leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… (AML). Our advancements in the understanding of epigenetics in these diseases have helped develop groundbreaking therapeutics that have changed the treatment landscape of MDS and AML, significantly improving outcomes. In this review we describe the most common epigenetic aberrations in MDS and AML, and current treatments that target mutations in epigenetic modifiers, as well as novel treatment combinations, from standard therapies to investigational treatments.

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