MYC proteins are major drivers of human tumorigenesis.592 For targeting MYC, Dicer-substrate siRNA (DsiRNA) formulated in EnCore lipid nanoparticles (DCR-MYC) has demonstrated efficacy in various tumor models in vivo.593,594 In one patient, DCR-MYC anticancer therapy was linked to thrombotic microangiopathy in the kidney.595 The patient ultimately died from cancer progression one year after DCR-MYC treatment was halted.595 Moreover, DCR-MYC is the first MYC-targeting siRNA to undergo human trials (NCT02110563). The gene discussed is MYC; the disease is cancer.