Target-stabilized base editors enable robust high-fidelity RNA editing

RNA base editing using engineered deaminases represents 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 devise 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 reduces transcriptome-wide edits while maintaining high on-target efficacy. Engineering gRNA for target RNA-induced conformational switching confines ADAR1d stabilization to intended editing sites, further enhancing editing precision. With structure-guided rational engineering of ADAR1d, RECODE efficiently corrects an Amyotrophic Lateral Sclerosis-relevant FUS mutation and installs a therapeutic mutation to Angptl3 in vivo, which mitigate FUS mislocalization to neuronal axons and lower plasma lipids, 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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Publication Details

Journal
Nature Communications
Published
2026-02-25
DOI
https://doi.org/10.1038/s41467-026-69835-w
Primary Topic
RNA regulation and disease
Type
article
Field-Weighted Citation Impact
0.00

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article

Target-stabilized base editors enable robust high-fidelity RNA editing

Zexuan Hong, Yunyi Lin, Yujing Zhang, Wenhui Liao et al.
Nature Communications
RNA regulation and disease
article

Target-stabilized base editors enable robust high-fidelity RNA editing

Zexuan Hong, Yunyi Lin, Yujing Zhang, Wenhui Liao, Zhonghua Lu, Qiwei Liu, Taian Liu, Yunping Lin, Jianqing Zhang, Lixin Yang, Wenhua Cao
article en

Abstract

RNA base editing using engineered deaminases represents 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 devise 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 reduces transcriptome-wide edits while maintaining high on-target efficacy. Engineering gRNA for target RNA-induced conformational switching confines ADAR1d stabilization to intended editing sites, further enhancing editing precision. With structure-guided rational engineering of ADAR1d, RECODE efficiently corrects an Amyotrophic Lateral Sclerosis-relevant FUS mutation and installs a therapeutic mutation to Angptl3 in vivo, which mitigate FUS mislocalization to neuronal axons and lower plasma lipids, 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.

Nature Communications
Shenzhen Maternity and Child Healthcare Hospital (CN), Shenzhen Institutes of Advanced Technology (CN), University of Chinese Academy of Sciences (CN), Southern Medical University (CN)
National Natural Science Foundation of China, Chinese Academy of Sciences, China Postdoctoral Science Foundation, Science, Technology and Innovation Commission of Shenzhen Municipality
Openalex Percentile: Top 43%
RNA regulation and disease
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