Loss of exosomes in progranulin-associated frontotemporal dementia

Neurobiol Aging. 2016 Apr:40:41-49. doi: 10.1016/j.neurobiolaging.2016.01.001. Epub 2016 Jan 7.

Abstract

Many cells of the nervous system have been shown to release exosomes, a subclass of secreted vesicles of endosomal origin capable of transferring biomolecules among cells: this transfer modality represents a novel physiological form of intercellular communication between neural cells. Herein, we demonstrated that progranulin (PGRN), a protein targeted to the classical secretory pathway, is also secreted in association with exosomes by human primary fibroblasts. Moreover, we demonstrated that null mutations in the progranulin gene (GRN), a major cause of frontotemporal dementia, strongly reduce the number of released exosomes and alter their composition. In vitro GRN silencing in SHSY-5Y cells confirmed a role of PGRN in the control of exosome release. It is believed that depletion of PGRN in the brain might cause neurodegeneration in GRN-associated frontotemporal dementia. We demonstrated that, along with shortage of the circulating PGRN, GRN null mutations alter intercellular communication. Thus, a better understanding of the role played by exosomes in GRN-associated neurodegeneration is crucial for the development of novel therapies for these diseases.

Keywords: Exosomes; Extracellular vesicles; GRN; Human primary fibroblasts; Null mutations; Progranulin.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Brain / pathology
  • Cells, Cultured
  • Exosomes / metabolism*
  • Female
  • Fibroblasts / metabolism*
  • Frontotemporal Dementia / genetics*
  • Frontotemporal Dementia / pathology
  • Frontotemporal Dementia / therapy
  • Gene Silencing
  • Humans
  • Intercellular Signaling Peptides and Proteins / genetics*
  • Intercellular Signaling Peptides and Proteins / metabolism*
  • Intercellular Signaling Peptides and Proteins / physiology
  • Male
  • Middle Aged
  • Molecular Targeted Therapy
  • Mutation
  • Progranulins

Substances

  • GRN protein, human
  • Intercellular Signaling Peptides and Proteins
  • Progranulins