Article
Synthetic introns enable splicing factor mutation-dependent targeting of cancer cells.
Nature biotechnology - 1 Jul 2022
North Khrystyna, Benbarche Salima, Liu Bo, Pangallo Joseph, Chen Sisi, Stahl Maximilian, Bewersdorf Jan Philipp, Stanley Robert F, Erickson Caroline, Cho Hana, Pineda Jose Mario Bello, Thomas James D, Polaski Jacob T, Belleville Andrea E, Gabel Austin M, Udy Dylan B, Humbert Olivier, Kiem Hans-Peter, Abdel-Wahab Omar, Bradley Robert K
Abstract excerpt
Many cancers carry recurrent, change-of-function mutations affecting RNA splicing factors. Here, we describe a method to harness this abnormal splicing activity to drive splicing factor mutation-dependent gene expression to selectively eliminate tumor cells. We engineered synthetic introns that were efficiently spliced in cancer cells bearing SF3B1 mutations, but unspliced in otherwise isogenic wild-type cells,...
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