
Seminars in Hematology, Год журнала: 2024, Номер unknown
Опубликована: Окт. 1, 2024
Myelodysplastic syndromes (MDS) represent a heterogeneous group of hematological disorders and are more commonly found in people over the age 60. MDS patients exhibit peripheral blood cytopenias carry an increased risk disease progression to acute myeloid leukemia (AML). Splicing factor mutations (including genes SF3B1, SRSF2, U2AF1, ZRSR2) early events identified than 50% cases. These cause aberrant pre-mRNA splicing impact pathophysiology. Emerging evidence shows that factor-mutant cells sensitive perturbations targeting spliceosome, aberrantly spliced and/or their regulated molecular pathways. This review summarizes current therapeutic strategies ongoing efforts for treatment MDS.
Язык: Английский