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MicroRNA-1224 (miR-1224) is an endogenous non-coding RNA, approximately 20–22 nucleotides in length, primarily functioning in the negative post-transcriptional regulation of gene expression by binding to the 3’ untranslated regions (3’UTRs) of target mRNAs[1][2]. It is located on human chromosome 3q27.1, originating from the last intron of the VWA5B2 gene, and is considered a “mirtron” (a microRNA processed from an intron without Drosha cleavage)[1]. miR-1224 has two mature forms: miR-1224-5p and miR-1224-3p, each participating in distinct regulatory roles. Extensive research supports its function as a tumor suppressor in diverse cancers (lung, gastric, glioma, osteosarcoma, bladder, colorectal, hepatocellular carcinoma, melanoma, and others); it inhibits cell proliferation, migration, invasion, promotes apoptosis and autophagy, and regulates key oncogenic pathways such as Wnt/β-catenin and EMT. Additionally, miR-1224 is implicated in other diseases, including acute liver failure and neurodegeneration. Its aberrant expression is associated with cancer development and prognosis, positioning it as a potential biomarker and therapeutic target[1][2]. Notes: - There are currently no approved drugs that directly modulate miR-1224; its role is as a biomarker and potential experimental target, but no drugs in clinical practice target this microRNA specifically[1][2]. - It is not a protein or classical drug receptor, enzyme, transporter, or ion channel, but a regulatory non-coding RNA; classification is best as "other" under molecular classifications[1][2]. - There are no known canonical safety liabilities specific to miR-1224, but gene therapy or oligonucleotide therapeutics targeting microRNA(s) in general should be approached cautiously due to the broad impact on gene networks.
Gene silencing via binding to the 3’ untranslated region (3’UTR) of mRNA, leading to mRNA degradation or translational repression
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