Abstract
RNA methylation and the machinery that regulates or 'reads' its expression has recently been implicated in the pathogenesis of acute myeloid leukemia (AML) and other hematological malignancies. Modulation of these epigenetic marks has started to become a reality as several companies around the world seek to leverage this knowledge therapeutically in the clinic. While the bases of observed activity in AML have been described by numerous groups, the exact context in which these therapies will ultimately be used remains to be properly determined. Although context is likely to be of great importance here, a more 'global' mechanism of action might allow for more widespread applicability to multiple diseases or disease subtypes. In other areas such as the myelodysplastic and other pre-leukemic syndromes, data remains sparse. Ongoing work is needed to determine whether therapeutic modulation of RNA modifications is a viable and biologically plausible approach in these cases. Regardless of the outcomes, this is an exciting era for 'epitranscriptomics' as we navigate a pathway forwards. Herein I describe the current knowledge around RNA methylation and hematological malignancies including some of the relevant questions that are yet to be answered.
Published Version
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