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  • Seiler, Michael; Yoshimi, Akihide; Darman, Rachel; Chan, Betty; Keaney, Gregg; Thomas, Michael; Agrawal, Anant A; Caleb, Benjamin; Csibi, Alfredo; Sean, Eckley; Fekkes, Peter; Karr, Craig; Klimek, Virginia; Lai, George; Lee, Linda; Kumar, Pavan; Lee, Stanley Chun-Wei; Liu, Xiang; Mackenzie, Crystal; Meeske, Carol; Mizui, Yoshiharu; Padron, Eric; Park, Eunice; Pazolli, Ermira; Peng, Shouyong; Prajapati, Sudeep; Taylor, Justin; Teng, Teng; Wang, John; Warmuth, Markus; Yao, Huilan; Yu, Lihua; Zhu, Ping; Abdel-Wahab, Omar; Smith, Peter G; Buonamici, Silvia

    Nature medicine, 04/2018, Letnik: 24, Številka: 4
    Journal Article

    Genomic analyses of cancer have identified recurrent point mutations in the RNA splicing factor-encoding genes SF3B1, U2AF1, and SRSF2 that confer an alteration of function. Cancer cells bearing these mutations are preferentially dependent on wild-type (WT) spliceosome function, but clinically relevant means to therapeutically target the spliceosome do not currently exist. Here we describe an orally available modulator of the SF3b complex, H3B-8800, which potently and preferentially kills spliceosome-mutant epithelial and hematologic tumor cells. These killing effects of H3B-8800 are due to its direct interaction with the SF3b complex, as evidenced by loss of H3B-8800 activity in drug-resistant cells bearing mutations in genes encoding SF3b components. Although H3B-8800 modulates WT and mutant spliceosome activity, the preferential killing of spliceosome-mutant cells is due to retention of short, GC-rich introns, which are enriched for genes encoding spliceosome components. These data demonstrate the therapeutic potential of splicing modulation in spliceosome-mutant cancers.