Article
Eupatilin rescues ciliary transition zone defects to ameliorate ciliopathy-related phenotypes.
The Journal of clinical investigation - 1 Aug 2018
Kim Yong Joon, Kim Sungsoo, Jung Yooju, Jung Eunji, Kwon Ho Jeong, Kim Joon
Abstract excerpt
Ciliopathies are clinically overlapping genetic disorders involving structural and functional abnormalities of cilia. Currently, there are no small-molecule drugs available to treat ciliary defects in ciliopathies. Our phenotype-based screen identified the flavonoid eupatilin and its analogs as lead compounds for developing ciliopathy medication. CEP290, a gene mutated in several ciliopathies, encodes a protein...
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