ArticleAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2023
Develop a Compact RNA Base Editor by Fusing ADAR with Engineered EcCas6e.
Article in Advanced science (Weinheim, Baden-Wurttemberg, Germany), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed.
- Target-stabilized base editors enable robust high-fidelity RNA editing.Nature communications · 2026Article
- Emerging trends in gene and cell therapy: CRISPR in DNA editing and beyond.Cell reports. Medicine · 2026Review
- A versatile type VI CRISPR-based approach for targeted mGenome research · 2026Article
- Targeted RNA base editing for therapeutic: mechanisms and advances.Pharmaceutical science advances · 2025Review
- RECODE: a programmable guide-free C-to-U RNA editing tool.Nucleic acids research · 2025Article
- Review
- TIGR-Tas and the Expanding Universe of RNA-Guided Genome Editing Systems: A New Era Beyond CRISPR-Cas.Genes · 2025Review
- ADARs: pleiotropy in function, versatility in application.Nucleic acids research · 2025Review
- Enhancing Specificity, Precision, Accessibility, Flexibility, and Safety to Overcome Traditional CRISPR/Cas Editing Challenges and Shape Future Innovations.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Review
- Programming ADAR-recruiting hairpin RNA sensor to detect endogenous molecules.Nucleic acids research · 2025Article
- EcCas6e-based antisense crRNA for gene repression and RNA editing in microorganisms.Nucleic acids research · 2024Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Expanding RNA editing toolkit using an IDR-based strategy.Molecular therapy. Nucleic acids · 2024Article
- RNA editing enzymes: structure, biological functions and applications.Cell & bioscience · 2024Review
- Breaking genetic shackles: The advance of base editing in genetic disorder treatment.Frontiers in pharmacology · 2024Review
- RNA base editing therapy cures hearing loss induced by OTOF gene mutation.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Develop a Compact RNA Base Editor by Fusing ADAR with Engineered EcCas6e.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2023Article
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Authors and funding
13 authors.
Funding
Abstract
Catalytically inactive CRISPR-Cas13 (dCas13)-based base editors can achieve the conversion of adenine-to-inosine (A-to-I) or cytidine-to-uridine (C-to-U) at the RNA level, however, the large size of dCas13 protein limits its in vivo applications. Here, a compact and efficient RNA base editor (ceRBE) is reported with high in vivo editing efficiency. The larger dCas13 protein is replaced with a 199-amino acid EcCas6e protein, derived from the Class 1 CRISPR family involved in pre-crRNA processing, and conducted optimization for toxicity and editing efficiency. The ceRBE efficiently achieves both A-to-I and C-to-U base editing with low transcriptome off-target in HEK293T cells. The efficient repair of the DMD Q1392X mutation (68.3±10.1%) is also demonstrated in a humanized mouse model of Duchenne muscular dystrophy (DMD) after AAV delivery, achieving restoration of expression for gene products. The study supports that the compact and efficient ceRBE has great potential for treating genetic diseases.
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