Mechanisms of Neurological Dysfunction in GOSR2 Progressive Myoclonus Epilepsy, a Golgi SNAREopathy

Neuroscience. 2019 Nov 10:420:41-49. doi: 10.1016/j.neuroscience.2019.03.057. Epub 2019 Apr 4.

Abstract

Successive fusion events between transport vesicles and their target membranes mediate trafficking of secreted, membrane- and organelle-localised proteins. During the initial steps of this process, termed the secretory pathway, COPII vesicles bud from the endoplasmic reticulum (ER) and fuse with the cis-Golgi membrane, thus depositing their cargo. This fusion step is driven by a quartet of SNARE proteins that includes the cis-Golgi t-SNARE Membrin, encoded by the GOSR2 gene. Mis-sense mutations in GOSR2 result in Progressive Myoclonus Epilepsy (PME), a severe neurological disorder characterised by ataxia, myoclonus and seizures in the absence of significant cognitive impairment. However, given the ubiquitous and essential function of ER-to-Golgi transport, why GOSR2 mutations cause neurological dysfunction and not lethality or a broader range of developmental defects has remained an enigma. Here we highlight new work that has shed light on this issue and incorporate insights into canonical and non-canonical secretory trafficking pathways in neurons to speculate as to the cellular and molecular mechanisms underlying GOSR2 PME. This article is part of a Special Issue entitled: SNARE proteins: a long journey of science in brain physiology and pathology: from molecular.

Keywords: GOSR2; Golgi; Membrin; progressive myoclonus epilepsy; secretory pathway.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Golgi Apparatus / metabolism
  • Golgi Apparatus / pathology
  • Humans
  • Mutation
  • Myoclonic Epilepsies, Progressive / genetics*
  • Myoclonic Epilepsies, Progressive / metabolism
  • Myoclonic Epilepsies, Progressive / physiopathology*
  • Neurons / metabolism
  • Neurons / pathology
  • Protein Transport / genetics*
  • Qb-SNARE Proteins / genetics*
  • Qb-SNARE Proteins / metabolism*

Substances

  • GOSR2 protein, human
  • Qb-SNARE Proteins