Gut-seeded α-synuclein fibrils promote gut dysfunction and brain pathology specifically in aged mice

Nat Neurosci. 2020 Mar;23(3):327-336. doi: 10.1038/s41593-020-0589-7. Epub 2020 Feb 17.

Abstract

Parkinson's disease is a synucleinopathy that is characterized by motor dysfunction, death of midbrain dopaminergic neurons and accumulation of α-synuclein (α-Syn) aggregates. Evidence suggests that α-Syn aggregation can originate in peripheral tissues and progress to the brain via autonomic fibers. We tested this by inoculating the duodenal wall of mice with α-Syn preformed fibrils. Following inoculation, we observed gastrointestinal deficits and physiological changes to the enteric nervous system. Using the AAV-PHP.S capsid to target the lysosomal enzyme glucocerebrosidase for peripheral gene transfer, we found that α-Syn pathology is reduced due to the increased expression of this protein. Lastly, inoculation of α-Syn fibrils in aged mice, but not younger mice, resulted in progression of α-Syn histopathology to the midbrain and subsequent motor defects. Our results characterize peripheral synucleinopathy in prodromal Parkinson's disease and explore cellular mechanisms for the gut-to-brain progression of α-Syn pathology.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Brain / pathology*
  • Digestive System Diseases / pathology*
  • Duodenum / pathology
  • Enteric Nervous System / pathology
  • Glucosylceramidase / biosynthesis
  • Glucosylceramidase / genetics
  • Mesencephalon / pathology
  • Mice
  • Mice, Inbred C57BL
  • Movement Disorders / etiology
  • Movement Disorders / pathology
  • Nerve Fibers / pathology
  • Nociception
  • Nodose Ganglion / pathology
  • Synucleinopathies / metabolism*
  • Synucleinopathies / pathology*

Substances

  • Glucosylceramidase