RAIDD mutations underlie the pathogenesis of thin lissencephaly (TLIS)

PLoS One. 2018 Oct 3;13(10):e0205042. doi: 10.1371/journal.pone.0205042. eCollection 2018.

Abstract

Abnormal regulation of caspase-2-mediated neuronal cell death causes neurodegenerative diseases and defective brain development. PIDDosome is caspase-2 activating complex composed of PIDD, RAIDD, and caspase-2. Recent whole-exome sequencing study showed that the RAIDD mutations in the death domain (DD), including G128R, F164C, R170C, and R170H mutations, cause thin lissencephaly (TLIS) by reducing caspase-2-mediated neuronal apoptosis. Given that the molecular structure of the RAIDD DD:PIDD DD complex is available, in this study, we analyzed the molecular mechanisms underlying TLIS caused by the RAIDD TLIS variants by performing mutagenesis and biochemical assays.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • CRADD Signaling Adaptor Protein / chemistry
  • CRADD Signaling Adaptor Protein / genetics*
  • CRADD Signaling Adaptor Protein / metabolism
  • Death Domain Receptor Signaling Adaptor Proteins / metabolism
  • Humans
  • Lissencephaly / etiology*
  • Lissencephaly / genetics*
  • Models, Molecular
  • Mutagenesis, Site-Directed
  • Point Mutation
  • Protein Domains

Substances

  • CRADD Signaling Adaptor Protein
  • Death Domain Receptor Signaling Adaptor Proteins

Grants and funding

This study was supported by the Basic Science Research Program through the National Research Foundation of Korea (NRF) of the Ministry of Education, Science and Technology (NRF-2017M3A9D8062960 and NRF-2018R1A2B2003635) and a grant from the Korea Healthcare Technology R&D Project, Ministry of Health & Welfare, Republic of Korea (HI17C0155).