Proof of concept about the suitability of MCPH genes as possible therapeutic targets has already been reported for ASPM, the gene mutated at the highest frequency in MCPH patients, as well as for KIF14 and CDK6. ASPM loss has been found to arrest the proliferation of glioma stem cells [38], to radiosensitize GBM cell lines [39], and to reduce tumor growth in MB mouse models [32] by increasing DNA double strand break (DSB) accumulation [32,39]. The gene discussed is ASPM; the disease is neoplasm.