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Aptamer 1-MNV-siTriple is a preclinical targeted RNA therapeutic being developed by the Mayo Clinic for the treatment of cholangiocarcinoma (CCA). The therapeutic platform consists of milk-derived nanovesicles (MNVs) decorated with a novel, highly specific DNA aptamer ('Aptamer 1') that targets CCA cells. The MNVs are loaded with a combination of three small interfering RNAs (siRNAs), collectively referred to as 'siTriple', which target Yes-associated protein 1 (Yap), WW domain-containing transcription regulator protein 1 (Wwtr1/TAZ), and tyrosine-protein kinase Lck (Lck). By knocking down these three key genes involved in CCA pathobiology, Aptamer 1-MNV-siTriple reduces tumor cell proliferation and enhances apoptosis, offering a precise, targeted gene therapy approach with minimal off-target effects on adjacent healthy liver tissue.
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