Clinical development of demethylating agents in hematology

Shyamala C. Navada, Juliane Steinmann, Michael Lübbert, Lewis R. Silverman

Research output: Contribution to journalReview articlepeer-review

120 Scopus citations


The term epigenetics refers to the heritable changes in gene expression that are not associated with a change in the actual DNA sequence. Epigenetic dysregulation is linked to the pathogenesis of a number of malignancies and has been studied extensively in myelodysplastic syndromes and acute myeloid leukemia. DNA methylation is frequently altered in cancerous cells and likely results in transcriptional silencing of tumor suppressor genes. Re-expression of these genes by inhibition of the DNA methyltransferases has been successful in the treatment of benign and malignant disease. In this Review, we discuss the clinical development of demethylating agents in hematology, with a focus on azacitidine and decitabine.

Original languageEnglish
Pages (from-to)40-46
Number of pages7
JournalJournal of Clinical Investigation
Issue number1
StatePublished - 2 Jan 2014


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