H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers.

doi:10.1038/nm.4493

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Article

H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers.

Nature medicine - 1 May 2018

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

Abstract excerpt

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...

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PMID: 29457796
DOI: 10.1038/nm.4493
PMCID: PMC6730556

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SRSF2

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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