Article
Splicing modulators impair DNA damage response and induce killing of cohesin-mutant MDS and AML.
Science translational medicine - 3 Jan 2024
Wheeler Emily C, Martin Benjamin J E, Doyle William C, Neaher Sofia, Conway Caroline A, Pitton Caroline N, Gorelov Rebecca A, Donahue Melanie, Jann Johann C, Abdel-Wahab Omar, Taylor Justin, Seiler Michael, Buonamici Silvia, Pikman Yana, Garcia Jacqueline S, Belizaire Roger, Adelman Karen, Tothova Zuzana
Abstract excerpt
Splicing modulation is a promising treatment strategy pursued to date only in splicing factor-mutant cancers; however, its therapeutic potential is poorly understood outside of this context. Like splicing factors, genes encoding components of the cohesin complex are frequently mutated in cancer, including myelodysplastic syndromes (MDS) and secondary acute myeloid leukemia (AML), where they are associated with...
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