LRRK2 kinase activity regulates GCase level and enzymatic activity differently depending on cell type in Parkinson’s disease

Output Details

Preprint September 28, 2021

Published July 18, 2022

Leucine-rich repeat kinase 2 (LRRK2) is a kinase involved in different cellular functions, including autophagy, endolysosomal pathways, and immune function. Mutations in LRRK2 cause autosomal-dominant forms of Parkinson’s disease (PD). Heterozygous mutations in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase (GCase), are the most common genetic risk factors for PD. Moreover, GCase function is altered in idiopathic PD and in other genetic forms of the disease. Recent work suggests that LRRK2 kinase activity can regulate GCase function. However, both a positive and a negative correlation have been described. To gain insights into the impact of LRRK2 on GCase, we performed a comprehensive analysis of GCase levels and activity in complementary LRRK2 models, including (i) LRRK2 G2019S knock in (GSKI) mice, (ii) peripheral blood mononuclear cell (PBMCs), plasma, and fibroblasts from PD patients carrying LRRK2 G2019S mutation, (iii) patient iPSCs-derived neurons; (iv) endogenous and overexpressed cell models. In some of these models we found a positive correlation between the activities of LRRK2 and GCase, which was further confirmed in cell lines with genetic and pharmacological manipulation of LRRK2 kinase activity. GCase protein level is reduced in GSKI brain tissues and in G2019S iPSCs-derived neurons, but increased in fibroblasts and PBMCs from patients, suggesting cell-type-specific effects. Overall, our study indicates that LRRK2 kinase activity affects both the levels and the catalytic activity of GCase in a cell-type-specific manner, with important implications in the context of therapeutic application of LRRK2 inhibitors in GBA1-linked and idiopathic PD.
Tags
  • Electron microscopy
  • GBA (Glucocerebrosidase)
  • GCase activity
  • iPSCs (Induced pluripotent stem cells)
  • LRRK2
  • Non-iPSC
  • Original Research

Meet the Authors

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    Maria Kedariti

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    Emanuele Frattini

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    Pascale Baden

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    Susanna Cogo

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    Laura Civiero

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    Elena Ziviani

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    Massimo Aureli

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    Alice Kaganovich

  • Mark Cookson, PhD

    National Institute on Aging

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    Leonidas Stefanis

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    Matthew Surface

  • Michela Deleidi, MD, PhD

    Co-PI (Core Leadership): Team Schapira

    Institut Imagine

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    Alessio Di Fonzo

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    Roy N. Alcalay

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    Hardy Rideout

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    Elisa Greggio

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    Nicoletta Plotegher