H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers Michael Seiler , Akihide Yoshimi , Rachel Darman , Betty Chan , Gregg Keaney , Michael Thomas , Anant A Agrawal , Benjamin Caleb , Alfredo Csibi , Eckley Sean , Peter Fekkes , Craig Karr , Virginia Klimek , George Lai , Linda Lee , Pavan Kumar , Stanley Chun-Wei Lee , Xiang Liu , Crystal Mackenzie , Carol Meeske , Yoshiharu Mizui , Eric Padron , Eunice Park , Ermira Pazolli , Shouyong Peng , Sudeep Prajapati , Justin Taylor , Teng Teng , John Wang , Markus Warmuth , Huilan Yao , Lihua Yu , Ping Zhu , Omar Abdel-Wahab , Peter G Smith , Silvia Buonamici Nature Medicine（2018）
摘要
The small molecule H3B-8800 selectively modulates RNA splicing to preferentially kill tumor cells bearing mutations in genes encoding spliceosome components.
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