Target-Stabilized Base Editors Enable Robust High-Fidelity RNA Editing
Taian LiuiD, Yunping Lin, Qiwei LiuiD, Wenhui Liao, Yunyi Lin, Yujing Zhang, Jianqing Zhang, Wenhua Cao, Lixin YangiD, Zexuan HongiD, Zhonghua LuiD
Abstract
RNA base editing using engineered deaminases expands target compatibility, offering a powerful tool to correct mutations at the RNA level. However, widespread off-target effects, primarily arising from dissociated free deaminases, remain a significant challenge. Here, we devised the RECODE (RNA editing with conditionally stable and enhanced ADAR1 deaminase variants) system, which employs designer degron-tagged ADAR1 deaminase (ADAR1d) with guide RNA (gRNA)-regulated stability. By promoting degradation of gRNA-unbound ADAR1d, RECODE markedly reduced transcriptome-wide edits while maintaining high on-target efficacy. Engineering gRNA for target RNA-induced conformational switching restricts ADAR1d stabilization, further enhancing editing precision. With structure-guided rational engineering of ADAR1d, RECODE achieved editing efficiencies of 90.3% and 58.8% for an Amyotrophic Lateral Sclerosis-relevant FUS mutation in vitro and in vivo, respectively. These findings establish RECODE as a highly stringent and efficient RNA editing technology and underscore a general principle for enhancing the specificity of RNA-guided protein effectors.
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