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MicroRNA-125b-5p is a highly conserved small non-coding RNA that plays a critical role in the post-transcriptional regulation of gene expression by binding to the 3'-untranslated regions (UTRs) of target mRNAs [miRBase, 2024; PubMed: 21901117]. It acts as a master regulator of various biological processes, including cell differentiation, proliferation, and the suppression of apoptosis by targeting pro-apoptotic genes like BAK1 and p53 [PubMed: 19833694]. In the context of human disease, MicroRNA-125b-5p is notably associated with hematological malignancies, particularly acute myeloid leukemia (AML), where its overexpression promotes leukemogenesis [PubMed: 20453308]. It also exhibits complex roles in solid tumors, functioning as either an oncogene or a tumor suppressor depending on the tissue type [PubMed: 23624835]. Beyond oncology, this miRNA is implicated in neurodegenerative conditions such as Alzheimer’s disease and chronic inflammatory responses [PubMed: 22761664]. Therapeutic development targeting MicroRNA-125b-5p primarily involves antisense oligonucleotides (antagomirs) to block its function in cancer or synthetic mimics to restore its levels in deficient states [PubMed: 26162310]. However, clinical translation is currently limited by the complexities of systemic delivery and the risk of off-target gene silencing across the miRNA's broad regulatory network [PubMed: 24703157].
MicroRNA-125b-5p regulates gene expression by binding to the seed sequence within the 3' UTR of target mRNAs, leading to mRNA cleavage or translational inhibition via the RNA-induced silencing complex (RISC) [PubMed: 21544161].
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