Engineered pistol ribozymes selectively target KRAS G12V with enhanced efficacy by capping modification.

Abstract excerpt

In this study, we rationally engineered a previously characterized pistol ribozyme to selectively target the KRAS p.G12V mutation with high specificity and efficiency. The pseudoknot-induced compact folding structure of the pistol ribozyme provides three-dimensional structural recognition of the KRAS substrate, distinguishing it from siRNA and ASO, which rely solely on primary sequence complementarity. This...

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