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

C Challis, A Hori, TR Sampson, BB Yoo… - Nature …, 2020 - nature.com
C Challis, A Hori, TR Sampson, BB Yoo, RC Challis, AM Hamilton, SK Mazmanian
Nature Neuroscience, 2020nature.com
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 …
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.
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