EpCAM-targeted aptamer-small interfering RNA

EpCAM-targeted aptamer-small interfering RNA (EpCAM-AsiC) is a nucleic acid-based therapeutic platform designed for the precision delivery of small interfering RNA (siRNA) to cancer cells. The platform utilizes an RNA aptamer that specifically binds to the epithelial cell adhesion molecule (EpCAM), a tumor-associated antigen frequently overexpressed in epithelial carcinomas and cancer stem cells. This aptamer is covalently or non-covalently linked to an siRNA payload. Upon binding to EpCAM on the cell surface, the chimera is internalized via receptor-mediated endocytosis, allowing the siRNA to escape the endosome and enter the RNA-induced silencing complex (RISC). This leads to the sequence-specific degradation of target mRNAs, such as those encoding PLK1, PD-L1, or mismatch repair proteins like MLH1 and MSH2. By combining the high specificity of aptamers with the potent gene-silencing capabilities of siRNA, this platform aims to overcome the delivery challenges of RNA therapeutics while minimizing systemic toxicity. Preclinical studies have explored its utility in treating various epithelial malignancies, including triple-negative breast cancer and colorectal cancer, often as a strategy to sensitize "cold" tumors to immune checkpoint inhibitors.

Other names

aptamer-siRNA chimerasEpCAM-targeted aptamer-siRNAsEpCAM-AsiCs