Clinically, early experiences with SINEs in AML and myelodysplastic syndromes (MDS) demonstrate on-target pharmacodynamic activity (nuclear re-accumulation of p53, downregulation of HOX/MEIS) that correlates with restored immune function—including increased infiltration of cytotoxic T cells into the bone marrow and reduced PD-L1 expression on blasts (Glaviano et al., 2025). This evidence concerns the gene CD274 and myelodysplastic syndrome.