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Subcellular localization

Ciliary associated gene, Cytosol

Functional category

• Small GTPases

Function

Primary cilia are antennae-like structures on cells that are important for early development and healthy cell function. Defects in primary cilia can cause inherited diseases called ciliopathies. Ciliopathies often cause fluid-filled sacs, called cysts, that are a major cause of kidney disease and failure. There is currently one drug licensed to slow kidney disease progression, but it is poorly tolerated in patients. Therefore, new drugs are needed. In this study, we used screening assays to identify potential drugs and their targets that are effective in promoting the formation of primary cilia. Our results identified ROCK2 (Rho-associated coiled-coil-containing protein kinase 2), an inhibitor of protein signalling, as a key mediator of cilium function. These findings suggest that drugs that specifically target ROCK2 could be a potential treatment option for cystic kidney disease(40253509).