MicroRNAs as Therapeutic Targets

MicroRNAs (miRNAs) are evolutionarily conserved class of small, regulatory RNAs that negatively regulate gene expression, and control multiple cellular processes involved in cancer initiation, progression, and metastasis. Extensive research in the past decade has identified vital role of miRNAs in hematopoiesis and leukemogenesis, with miRNAs functioning as oncogenes or tumor suppressors. Several miRNAs are extensively dysregulated in acute myeloid leukemia (AML), and their expression patterns are useful diagnostic and prognostic markers in AML. Furthermore, miRNA expression patterns have been associated with specific cytogenetic and molecular aberrations in AML subsets. Based on these observations, miRNAs have emerged as promising therapeutic tools for AML. In this chapter, we focus on the roles of miRNAs in AML tumorigenesis, their widespread deregulation, the rationale and strategies for their use as AML therapeutic targets, and the advantages and current challenges to their use in the clinic.