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FKBP51 siRNA is a small interfering RNA (siRNA) designed to silence the expression of the FK506-binding protein 51 (FKBP51), a co-chaperone protein that plays a significant role in melanoma plasticity and tumor aggressiveness. FKBP51 has been identified as a positive regulator of the melanoma cancer stem cell (CSC) phenotype and epithelial-to-mesenchymal transition (EMT) traits. By utilizing RNA interference to knock down FKBP51 at the transcriptional level, this therapeutic approach aims to inhibit the genetic programs that sustain melanoma stemness and invasiveness. Preclinical studies in animal models have demonstrated that systemic administration of FKBP51 siRNA can significantly reduce organ colonization and prevent metastasis in lungs and liver, as evidenced by qualitative and quantitative PET/CT imaging and histological examination. The research was primarily conducted by investigators at the University Federico II and the National Research Council (CNR) in Naples, Italy.
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