Target-Stabilized Base Editors Enable Robust High-Fidelity RNA Editing
Liu, T.; Lin, Y.; Liu, Q.; Liao, W.; Zhang, Y.; Cao, W.; Lin, Y.; Yang, L.; Hong, Z.; Lu, Z.
Show 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.
Matching journals
The top 3 journals account for 50% of the predicted probability mass.
Similar papers in this journal
Similar papers in this journal
- PAX3-FOXO1 coordinates enhancer architecture, eRNA transcription, andRNA polymerase pause release at select gene targets 98%
- Circadian PERIOD proteins regulate TC-DSB repair through anchoring to the nuclear envelope 97%
- TRIM24 directs replicative stress responses to maintain ALT telomeres via chromatin signaling 96%
Similar papers in this journal
"Similar papers" are the closest papers from that journal in the model's embedding space. They show what the match is built on, but the ranking comes mostly from a classifier over the whole training set, not from these examples alone.