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Synthetic miR-142 is an RNA therapy designed to restore the function of the microRNA miR-142, which is a short non-coding RNA molecule that regulates gene expression by downregulating target messenger RNAs (mRNAs), leading to mRNA degradation or translational inhibition. A deficit or downregulation of endogenous miR-142 has been linked to the progression of myeloproliferative neoplasms (MPNs) to secondary acute myeloid leukemia (sAML), as well as other hematological malignancies like acute myeloid leukemia (AML), lymphomas, and acute lymphoblastic leukemia (ALL). By re-establishing normal miR-142 levels, this therapy aims to normalize gene regulatory networks and signaling pathways involved in hematopoiesis and cancer progression. City of Hope is developing a synthetic miR-142 therapy for myeloproliferative neoplasms and their progression to sAML.
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