Genetic Modeling of ELP1-Associated Sonic Hedgehog Medulloblastoma Identifies MDM2 As a Selective Therapeutic Target

Genetically engineered mice mimicking heterozygous elongator acetyltransferase complex subunit 1 (Elp1) loss-of-function seen in affected germline carriers exhibit hallmark features of premalignancy in cerebellar granule neuron progenitors.