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Aprinocarsen is a synthetic phosphorothioate oligodeoxynucleotide that functions as an antisense molecule. It specifically inhibits the expression of protein kinase C-alpha (PKC-α) by hybridizing to its 3'-untranslated region mRNA, leading to RNase H-mediated cleavage of the hybridized mRNA and reduced PKC-α protein levels. This mechanism regulates cell differentiation and proliferation, demonstrating anti-tumor activity in various human tumor cell lines in preclinical models. It was initially developed by Isis Pharmaceuticals (now Ionis Pharmaceuticals) and later licensed to Eli Lilly & Co. for the treatment of human cancers.
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