Dysregulation of MDM2-mediated p53 ubiquitination in autism mouse models

  • Awarded: 2014
  • Award Type: Explorer
  • Award #: 336605

Multiple autism spectrum disorders share similar neuronal deficits at the molecular and cellular levels. Understanding the mechanism underlying those deficits helps researchers identify valuable targets for therapeutic development.

Nien-Pei Tsai tested two mouse models: the fragile X syndrome mouse model and the PTEN conditional knockout mouse model. In his preliminary work, he found that one of the protein modification pathways, protein ubiquitination, is dysregulated in a similar way in both mouse models.

Based on that work, Tsai predicted that this pathway is critical for certain autism symptoms and proposed to study this particular pathway with two specific goals. He aims to determine how this pathway is molecularly dysregulated. He also plans to correct this pathway pharmacologically and test whether certain neuronal deficits in the autism mouse models can be corrected. The data obtained from this project could benefit the autism field and open new avenues for future basic and clinical studies.

Subscribe to our newsletter and receive SFARI funding announcements and news