MDM2/p53轴功能障碍与早衰有关。
Dysfunction of the MDM2/p53 axis is linked to premature aging.
作者信息
Lessel Davor, Wu Danyi, Trujillo Carlos, Ramezani Thomas, Lessel Ivana, Alwasiyah Mohammad K, Saha Bidisha, Hisama Fuki M, Rading Katrin, Goebel Ingrid, Schütz Petra, Speit Günter, Högel Josef, Thiele Holger, Nürnberg Gudrun, Nürnberg Peter, Hammerschmidt Matthias, Zhu Yan, Tong David R, Katz Chen, Martin George M, Oshima Junko, Prives Carol, Kubisch Christian
机构信息
Institute of Human Genetics, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Department of Biological Sciences, Columbia University, New York, New York, USA.
出版信息
J Clin Invest. 2017 Oct 2;127(10):3598-3608. doi: 10.1172/JCI92171. Epub 2017 Aug 28.
The tumor suppressor p53, a master regulator of the cellular response to stress, is tightly regulated by the E3 ubiquitin ligase MDM2 via an autoregulatory feedback loop. In addition to its well-established role in tumorigenesis, p53 has also been associated with aging in mice. Several mouse models with aberrantly increased p53 activity display signs of premature aging. However, the relationship between dysfunction of the MDM2/p53 axis and human aging remains elusive. Here, we have identified an antiterminating homozygous germline mutation in MDM2 in a patient affected by a segmental progeroid syndrome. We show that this mutation abrogates MDM2 activity, thereby resulting in enhanced levels and stability of p53. Analysis of the patient's primary cells, genome-edited cells, and in vitro and in vivo analyses confirmed the MDM2 mutation's aberrant regulation of p53 activity. Functional data from a zebrafish model further demonstrated that mutant Mdm2 was unable to rescue a p53-induced apoptotic phenotype. Altogether, our findings indicate that mutant MDM2 is a likely driver of the observed segmental form of progeria.
肿瘤抑制因子p53是细胞应激反应的主要调节因子,通过一个自动调节反馈环受到E3泛素连接酶MDM2的严格调控。除了在肿瘤发生中已确立的作用外,p53还与小鼠衰老有关。几种p53活性异常增加的小鼠模型表现出早衰迹象。然而,MDM2/p53轴功能障碍与人类衰老之间的关系仍然难以捉摸。在这里,我们在一名患有节段性早衰综合征的患者中发现了MDM2中的一个抗终止纯合种系突变。我们表明,这种突变消除了MDM2的活性,从而导致p53水平和稳定性增强。对患者原代细胞、基因编辑细胞以及体外和体内分析证实了MDM2突变对p53活性的异常调节。来自斑马鱼模型的功能数据进一步表明,突变型Mdm2无法挽救p53诱导的凋亡表型。总之,我们的研究结果表明,突变型MDM2可能是观察到的节段性早衰形式的驱动因素。