Screening methods and pharmaceutically active compounds for neurodegenerative diseases

摘要

The invention describes methods for detecting compounds that target the auto-inhibitory mechanism of ATP13A2 protein or ATP13A1/3-5 isoforms leading to a modulation of ATPase activity, the method comprising the steps of: providing an assay system comprising ATP13A2 protein or ATP13A1/3-5 isoforms, and a source of ATP, adding a test compound to said assay system, wherein the test compound is not a phosphatidic acid (PA) and/or phosphatidylinositol(3,5)bisphosphate (PI(3,5)P2), determining whether the test compound modifies ATP hydrolysis, compared to a control assay without test compound, wherein a test compound which modifies ATP hydrolysis is identified as modulator of ATPase activity.