Constitutive activation of mTORC1 signaling induced by biallelic loss-of-function mutations in SZT2 underlies a discernible neurodevelopmental disease

PLoS One. 2019 Aug 20;14(8):e0221482. doi: 10.1371/journal.pone.0221482. eCollection 2019.

Abstract

There have been increasing number of reports of SZT2-related neurological diseases, the main symptoms of which are epilepsy, developmental delay, macrocephaly and a dysmorphic corpus callosum. SZT2 functions as a regulator of mechanistic target of rapamycin complex 1 (mTORC1) signaling in cultured human cell lines and mouse tissues. However, it remains to be determined whether mutations in SZT2 in human patients alter mTORC1 signaling. In this study, we aimed to investigate the functional consequence of biallelic SZT2 variants in Epstein-Barr virus-induced lymphoblastoid cell lines (LCLs) established from two patients with a typical SZT2-related neurodevelopmental disease. Increased phosphorylation of S6 kinase and S6 was identified in patient-derived cell lines under amino acid-starved condition, suggestive of constitutive hyperactivation of mTORC1 signaling. This result was validated by constitutive lysosomal localization of mTOR in patients' LCLs. Furthermore, patients' LCLs display an excessive response to slight amino acid stimulation. Our data suggest the loss-of-function nature of SZT2 mutations in the patients, and consequent hyperactivation of mTORC1 signaling in response to both amino acid starvation and stimulation in their LCLs. By these functional analyses, the pathogenicity of newly identified SZT2 variants can be determined, allowing for more detailed characterization of genotype-phenotype correlations.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Alleles*
  • Cell Line
  • Child, Preschool
  • Female
  • Humans
  • Loss of Function Mutation / genetics*
  • Lysosomes / metabolism
  • Male
  • Mechanistic Target of Rapamycin Complex 1 / metabolism*
  • Nerve Tissue Proteins / genetics*
  • Neurodevelopmental Disorders / genetics*
  • Signal Transduction*

Substances

  • Nerve Tissue Proteins
  • SZT2 protein, human
  • Mechanistic Target of Rapamycin Complex 1

Grants and funding

This study was supported by the Initiative on Rare and Undiagnosed Diseases from the Japanese Agency for Medical Research and Development (AMED) under the grant numbers JP18ek0109301 (YT, SS) and JP18ek0109280 (NM). There was no additional external funding received for this study.