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.
Authors
- Zexuan Hong
- Yunyi Lin
- Yujing Zhang
- Wenhui Liao
- Zhonghua Lu
- Qiwei Liu
- Taian Liu
- Yunping Lin
- Jianqing Zhang
- Lixin Yang
- Wenhua Cao
Institutions
- Shenzhen Maternity and Child Healthcare Hospital (CN)
- Shenzhen Institutes of Advanced Technology (CN)
- University of Chinese Academy of Sciences (CN)
- Southern Medical University (CN)
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
Funders
- National Natural Science Foundation of China
- Chinese Academy of Sciences
- China Postdoctoral Science Foundation
- Science, Technology and Innovation Commission of Shenzhen Municipality