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Convergent molecular pathways to inherited Parkinson's disease - PubMed

a day ago
  • #LRRK2
  • #Parkinson's disease
  • #GBA1
  • Pathogenic LRRK2 variants increase kinase activity, while GBA1 variants reduce lysosomal glucocerebrosidase activity, both linked to Parkinson's disease.
  • LRRK2 phosphorylates Rab GTPases, influencing cellular pathology through phosphorylation-specific effectors.
  • LRRK2 roles include promoting exocytosis of lysosome-related organelles and regulating primary cilia formation in neurons and astrocytes.
  • Loss of primary cilia or GBA1 deficiency impairs Hedgehog signaling, reducing neuroprotective factors for dopamine neurons.
  • LRRK2 inhibitors in mutant mice restore cilia and neuroprotective factor production, showing therapeutic potential.