VEXAS patient CD34+ cells exhibited significantly lower IC_50 values and higher rates of apoptosis upon TAK-243 exposure than cells from healthy donors or myelodysplastic syndrome (MDS) controls, suggesting a broad therapeutic window in which UBA1-mutant clones are selectively targeted while sparing normal hematopoiesis (72). The gene discussed is CD34; the disease is myelodysplastic syndrome.