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Cell division control protein 42 homolog (CDC42) is a highly conserved small GTPase belonging to the Rho family that acts as a molecular switch in various signaling pathways. It is primarily responsible for regulating the actin cytoskeleton, cell polarity, and vesicle trafficking (UniProt: P60953). The mRNA of CDC42 serves as the template for this protein and is a target for RNA-based therapeutic interventions. Overexpression or hyperactivation of CDC42 is linked to increased tumor cell proliferation, invasion, and metastasis in several cancers (PubMed: 30232358). Conversely, germline mutations in the CDC42 gene are the cause of Takenouchi-Kosaki syndrome, which involves developmental delays and hematological abnormalities (PubMed: 26437034). Therapeutic strategies targeting CDC42 mRNA include small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs) designed to induce mRNA degradation and reduce protein levels. These approaches are currently being explored in preclinical models to treat aggressive malignancies where CDC42 is a driver. A major challenge in targeting CDC42 mRNA is its ubiquitous expression and essential role in normal physiology, which can lead to significant toxicity if not targeted specifically to diseased tissues. Monitoring CDC42 mRNA levels can serve as a biomarker for disease progression and therapeutic efficacy in these contexts.
RNA interference (RNAi) or antisense-mediated mRNA degradation leading to reduced protein synthesis.
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