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ATPase phospholipid-transporting 10B (ATP10B) is a catalytic subunit of the P4-ATPase flippase complex, a class of enzymes responsible for the ATP-dependent translocation ("flipping") of specific phospholipids such as glucosylceramide and phosphatidylcholine from the outer to the inner leaflet of lysosomal membranes. ATP10B is predominantly localized in the endoplasmic reticulum, late endosome, and lysosomal membranes, and is especially expressed in brain regions such as the substantia nigra and basal ganglia, among others[1][3]. ATP10B maintains lysosomal membrane integrity and function, particularly in neurons[1][2]. Recessive loss-of-function mutations have been associated with increased risk for Parkinson’s disease by impairing lysosomal lipid export, leading to lysosomal dysfunction and neuronal cell death[2]. ATP10B is also linked to some forms of hereditary deafness and Waardenburg syndrome[1]. No drugs are currently known to target ATP10B directly.
No approved drugs known to directly target ATP10B. Loss-of-function mutations cause disrupted lysosomal export of glucosylceramide and phosphatidylcholine, contributing to disease pathology[2].
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