Dysfunction of the MDM2/p53 axis is linked to premature aging

J Clin Invest. 2017 Oct 2;127(10):3598-3608. doi: 10.1172/JCI92171. Epub 2017 Aug 28.

Abstract

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.

MeSH terms

  • Aging, Premature* / genetics
  • Aging, Premature* / metabolism
  • Animals
  • Apoptosis / genetics
  • Cell Line, Tumor
  • Disease Models, Animal
  • Germ-Line Mutation*
  • Humans
  • Proto-Oncogene Proteins c-mdm2* / genetics
  • Proto-Oncogene Proteins c-mdm2* / metabolism
  • Tumor Suppressor Protein p53* / genetics
  • Tumor Suppressor Protein p53* / metabolism
  • Zebrafish Proteins* / genetics
  • Zebrafish Proteins* / metabolism
  • Zebrafish* / genetics
  • Zebrafish* / metabolism

Substances

  • TP53 protein, human
  • Tumor Suppressor Protein p53
  • Zebrafish Proteins
  • MDM2 protein, human
  • Proto-Oncogene Proteins c-mdm2
  • mdm2 protein, zebrafish