Article
Allele-Specific RNA Knockdown with a Biologically Stable and Catalytically Efficient XNAzyme.
Journal of the American Chemical Society - 31 Mar 2021
Nguyen Kim, Wang Yajun, England Whitney E, Chaput John C, Spitale Robert C
Abstract excerpt
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...
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