Current ADAR-recruiting RNA editing systems suffer from limited efficacy and bystander editing. We developed a system in which the multivalent recruitment of endogenous ADAR1 p150 through its Zα domain enables up to 100% A-to-I editing in vitro and 84% in vivo, enabling precise correction of disease-associated mutations at the transcript level.
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References
Pfeiffer, L. S. & Stafforst, T. Precision RNA base editing with engineered and endogenous effectors. Nat. Biotechnol. 41, 1526–1542 (2023). A review that comprehensively surveys current RNA base-editing strategies, with a focus on harnessing endogenous ADAR enzymes.
Li, J. B. & Walkley, C. R. Leveraging genetics to understand ADAR1-mediated RNA editing in health and disease. Nat. Rev. ...