MDM4是致癌基因，编码蛋白是p53抑癌基因的负调控因子。p53的两个主要负调控因子，MDM2和MDM4，通过阻断其N-末端反式激活域发挥作用。此外，MDM4刺激MDM2催化的p53泛素化，导致其降解。与MDM2类似，MDM4在多种肿瘤类型（皮肤黑色素瘤、原发性乳腺肿瘤、视网膜母细胞瘤、Ewing肉瘤和胶质瘤）中失去调控。

MDM4 encodes a negative regulator of the p53 tumor suppressor. MDM4 is amplified or overexpressed in a diverse range of cancers resulting in aberrant p53 activity.The MDM4 gene encodes a negative regulator of the p53 tumor suppressor, the most frequently inactivated gene in human cancer. The two major negative regulators of p53, MDM2 and MDM4, function by blocking its N-terminal transactivation domain. In addition, MDM4 stimulates the MDM2-catalysed ubiquitination of p53 which leads to its degradation. Similar to MDM2, MDM4 is deregulated in several tumor types such as cutaneous melanoma, primary breast tumors, retinoblastomas, Ewing sarcomas and gliomas. In the overwhelming majority of cases, cells with deregulated MDM2 or MDM4 retain wild-type p53. Thus, inhibition of the p53 interaction with MDM2 or MDM4 is an attractive candidate strategy for treatment of cancer via the reactivation of p53.

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