Article

<i>SF3B1</i> -mutant mis-splicing of <i>UBA1</i> confers a targetable therapeutic vulnerability through UBA1 inhibition

2024-08-28

Abstract excerpt

SF3B1 mutation-driven myelodysplastic syndromes (MDS- SF3B1 ) arise due to somatic mutation in the splicing factor SF3B1 gene. SF3B1 mutations induce RNA mis-splicing and loss of expression of critical genes for erythropoiesis, leading to erythroid dysplasia and ultimately refractory anemia. The development of precision medicine approaches for MDS- SF3B1 is hampered by the complexity of the mis-splicing landsc...

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Literature Corpus work: f066bc3d-e5fa-5b4e-acd2-88edbb4841b1
DOI: 10.1101/2024.08.28.610114

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