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E7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial

9 Oct 2023

Abstract excerpt

Treatment options for relapsed and/or refractory (R/R) MDS and AML are limited. Mutations in genes encoding RNA splicing factors are encountered frequently in patients with AML and in up to 60% of MDS patients [ 1 , 2 ]. As splicing factor mutations are often mutually exclusive, splicing factor-mutant cells could be dependent on residual wild-type splicing for survival [ 3 , 4 ]. Targeting residual splicing...

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PMID: 37814121
DOI: 10.1038/s41375-023-02050-4

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