Abstract

Simple SummaryAlterations in primary RNA motifs and aberrant expression levels of non-coding RNA molecules have emerged as biomarkers of disease development and progression. Advances in antisense oligonucleotide (ASO) techniques and pharmacologic discoveries in targeting of RNA structures and RNA–protein interactions with small molecules open a new area in RNA therapeutics that may help in developing a next generation of anti-cancer drugs.RNA molecules are a source of phenotypic diversity and an operating system that connects multiple genetic and metabolic processes in the cell. A dysregulated RNA network is a common feature of cancer. Aberrant expression of long non-coding RNA (lncRNA), micro RNA (miRNA), and circular RNA (circRNA) in tumors compared to their normal counterparts, as well as the recurrent mutations in functional regulatory cis-acting RNA motifs have emerged as biomarkers of disease development and progression, opening avenues for the design of novel therapeutic approaches. This review looks at the progress, challenges and future prospects of targeting cis-acting and trans-acting RNA elements for leukemia diagnosis and treatment.

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