bioRxiv · 10.1101/465567
Long-term exposure to PFE-360 in the AAV-α-synuclein rat model: findings and implications
Abstract
Significance statementTreatment of Parkinsons disease is reliant on symptomatic treatments, without any option to slow or halt disease progression. Mutations in LRRK2 and -synuclein are known risk factors for Parkinsons disease. Presence of -synuclein aggregates at autopsies in both idiopathic and most G2019S cases is suggestive of a common disease pathogenesis. LRRK2 and -synuclein interaction is hypothesized to play a pivotal role in the pathological mechanisms and inhibitors of LRRK2 are investigated as novel disease modulatory treatments in the clinic. However, preclinical in vivo evidence of a beneficial effect of LRRK2 inhibition is mixed and limited. This study increases our understanding of LRRK2 as a mediator of neuronal dysfunction and the potential of LRRK2 as a promising target in PD.
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Andersen, M. A., Sotty, F., Jensen, P. H., Badolo, L., Jeggo, R., Smith, G. P., Christensen, K. V.. 2018-11-12. Long-term exposure to PFE-360 in the AAV-α-synuclein rat model: findings and implications. https://doi.org/10.1101/465567
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