Sensitivity to ATR-CHK1 pathway inhibition in MDS/AML is enhanced by SRSF2 mutations and reduced by RUNX1 loss.
SRSF2 mutations occur in 5-15% of acute myeloid leukemia (AML) and ~15% of myelodysplastic neoplasms (MDS), are enriched in elderly/secondary AML, and lack mutation-directed therapy. We aimed to identify vulnerabilities in MDS/AML with SRSF2 mutations. Ex vivo drug-sensitivity testing of bone marrow cells from AML pati...