Ontology highlight
ABSTRACT:
SUBMITTER: Seiler M
PROVIDER: S-EPMC6730556 | biostudies-literature | 2018 May
REPOSITORIES: biostudies-literature
Nature medicine 20180219 4
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 splice ...[more]