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Therapeutic targeting of allele-specific single nucleotide mutations in RNA is a major challenge in biology and medicine. Herein, we describe the utility of the XNAzyme X10-23 to knock down allele-specific mRNA sequences in cells. We demonstrate the value of this approach by targeting the "undruggable" mutation G12V in oncogenic KRAS. Our results demonstrate how catalytic XNAs could be employed to suppress the expression of mRNAs carrying disease-causing mutations that are difficult to target at the protein level with small molecule therapeutics.


Kim Nguyen, Yajun Wang, Whitney E England, John C Chaput, Robert C Spitale. Allele-Specific RNA Knockdown with a Biologically Stable and Catalytically Efficient XNAzyme. Journal of the American Chemical Society. 2021 Mar 31;143(12):4519-4523

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PMID: 33750115

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