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Icahn School of Medicine at Mount Sinai

21 Claremont Avenue
Apt 94
NEW YORK, NY 10029
United States

Leukemia is the most common cancer in children, accounting for almost 1 out of 3 cancers. This is also true in children with Down syndrome, a chromosomal abnormality caused by a third copy of chromosome 21. In particular, children with Down syndrome have a 150-fold increased risk of developing acute myeloid leukemia during the first years of their childhood. In fact, the first mutation occurs in the gene GATA1 during the development of the fetus at pregnancy.

Background

Children with familial RUNX1 disorder have a high risk of developing leukemia, which in most cases is hard to treat with current chemotherapy. Finding ways to prevent progression of leukemia is the most promising strategy towards the effective management of this disorder.

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