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Article

Targeting Aberrant Splicing in Myelodysplastic Syndromes: Biologic Rationale and Clinical Opportunity.

Hematology/oncology clinics of North America - 1 Apr 2020

Brunner Andrew M, Steensma David P

Abstract excerpt

Myelodysplastic syndromes are enriched for somatic mutations in the pre-mRNA splicing apparatus, with recurrent acquired mutations most commonly occurring in SF3B1, SRSF2, U2AF1, and ZRSR2. These mutations appear to be early events in the pathogenesis of disease, and, given their frequency and central role in leukemogenesis, are of interest as potential therapeutic targets. Clinical trials are exploring targets...

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Clonal Evolution
Disease Management
Molecular Targeted Therapy
Myelodysplastic Syndromes
Protein-Arginine N-Methyltransferases

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Synthetic Lethal Mutations

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Identifiers

PMID: 32089217
DOI: 10.1016/j.hoc.2019.10.003
PMCID: PMC7045909

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