ArticleNature cell biology2021
Eliminating base-editor-induced genome-wide and transcriptome-wide off-target mutations.
Article in Nature cell biology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 74 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
74 citing papers in PubMed, 97 citations in OpenAlex.
- Dense RNA motif modifications enable robust in vivo prime editing and enhance efficiencies of diverse editing systems.Nature biomedical engineering · 2026Article
- Engineered Transformer Base Editor with Enhanced Editing Efficiency.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Efficient in vivo cytosine base editing using virus-like particles with uracil DNA glycosylase inhibition.Nature biotechnology · 2026Article
- Engineered transformer base editor with unconstrained PAM requirements.Molecular therapy. Nucleic acids · 2026Article
- Article
- Multiplexed CRISPR base editing enables pulse-activated irreversible biocontainment of engineered bacteria.Nucleic acids research · 2026Article
- High-precision cytosine base editors by evolving nucleic-acid-recognition hotspots in deaminase.Nature biotechnology · 2026Article
- Regulated transformation system (RTS): sddi-mediated programmable shut-off and mode switching of base editors.Nucleic acids research · 2026Article
- Spatially concentrated adenine base editors efficiently correct PLP1 mutations in oligodendrocytes.Nucleic acids research · 2026Article
- Leveraging base excision repair for efficient adenine base editing of mitochondrial DNA.Nature biotechnology · 2026Article
- Specific and efficient RNA A-to-I editing through cleavage of an ADAR inhibitor.Nature biotechnology · 2026Article
- Emerging trends in gene and cell therapy: CRISPR in DNA editing and beyond.Cell reports. Medicine · 2026Review
- RNA editing: an emerging frontier in cancer therapy - explorations, opportunities, and challenges.International journal of surgery (London, England) · 2026Article
- CRISPR/Cas9 nickase-mediated cytosine base editing inMycology · 2026Article
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Research progress of base editing and prime editing tools based on the CRISPR/Cas system.Molecular therapy. Nucleic acids · 2025Review
- Effective in vivo RNA base editing via engineered cytidine deaminase APOBECs fused with PUF proteins.Nature communications · 2025Article
- Noncanonical target-strand cytosine base editing via engineered Un1Cas12f1 platform.Nature communications · 2025Article
- Cytotoxicity of activator expression in CRISPR-based transcriptional activation systems.Nature communications · 2025Article
14 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The fusion of CRISPR-Cas9 with cytidine deaminases leads to base editors (BEs) capable of programmable C-to-T editing, which has potential in clinical applications but suffers from off-target (OT) mutations. Here, we used a cleavable deoxycytidine deaminase inhibitor (dCDI) domain to construct a transformer BE (tBE) system that induces efficient editing with only background levels of genome-wide and transcriptome-wide OT mutations. After being produced, the tBE remains inactive at OT sites with the fusion of a cleavable dCDI, therefore eliminating unintended mutations. When binding at on-target sites, the tBE is transformed to cleave off the dCDI domain and catalyses targeted deamination for precise base editing. After delivery into mice through a dual-adeno-associated virus (AAV) system, the tBE system created a premature stop codon in Pcsk9 and significantly reduced serum PCSK9, resulting in a ~30-40% decrease in total cholesterol. The development of tBE establishes a highly specific base editing system and its in vivo efficacy has potential for therapeutic applications.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.