Recent advances in the cellular and molecular understanding of myelodysplastic syndromes: implications for new therapeutic approaches.

ABSTRACT: It has been more than 10 years since any new disease-modifying therapies have received regulatory approval for indications related to myelodysplastic syndromes (MDS). Advances in our collective biological understanding of MDS in the last decade, however, have made it possible to hope that effective therapeutics can be designed to improve MDS-associated cytopenias and patients' quality of life, and perhaps even delay clonal progression and extend survival. Classes of MDS-associated mutations and disordered biological pathways targeted by developmental therapeutics include the following: aberrant messenger RNA splicing, neomorphic enzymes in the citric acid cycle with oncogenic activity, overactivated tyrosine and serine-threonine kinases, epigenetic and chromatin remodeling alterations, abn

SUBMITTER: Brunner AM 

PROVIDER: S-EPMC6629038 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature