ArticleMolecular therapy : the journal of the American Society of Gene Therapy2022
A precise and efficient adenine base editor.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
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Who cites it
35 citing papers in PubMed.
- Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.Human gene therapy · 2026Review
- Rational selection of SpCas9 and variant-derived adenine base editors for human genome editing.Gene therapy · 2026Article
- Rational selection of SpCas9 and variant-derived adenine base editors for human genome editing.Gene therapy · 2026Article
- Continuous Evolution System Based on Toxin-Antitoxin System Combined with Base Deaminase Accelerates the In Vivo Modification of Target Proteins.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- sgRNA amount is a limiting factor in adenine base editing using RNA LNPs.Molecular therapy. Nucleic acids · 2026Article
- Design of Safe and Efficient Adenine Base Editors via Protein Language Model Screening for Osteoarthritis Treatment.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Therapeutic base editing alleviates restrictive cardiomyopathy.Cell reports. Medicine · 2026Article
- Efficient mitochondrial A-to-G base editors for the generation of mitochondrial disease models.Nature biotechnology · 2026Article
- ABE9 fused to SpRY Cas9 nickase enables precise generation of bystander free mouse models.Scientific reports · 2026Article
- Review
- In vivo base editing rescues ADPKD in a humanized mouse model.Nature communications · 2025Article
- Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa.Gene therapy · 2025Article
- A compact base editor rescues AATD-associated liver and lung disease in mouse models.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Correcting a patient-specific Rhodopsin mutation with adenine base editor in a mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- CRISPR-based functional genomics tools in vertebrate models.Experimental & molecular medicine · 2025Review
- Charting the development and engineering of CRISPR base editors: lessons and inspirations.Cell chemical biology · 2025Review
- Recent advances in therapeutic gene-editing technologies.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Precise genome editing process and its applications in plants driven by AI.Functional & integrative genomics · 2025Review
- A base editor for the long-term restoration of auditory function in mice with recessive profound deafness.Nature biomedical engineering · 2025Article
- Rational Design of Enhanced Nme2Cas9 and Nme2bioRxiv : the preprint server for biology · 2024Article
Corrections and comments
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Authors and funding
18 authors.
Funding
Abstract
Adenine base editors (ABEs) are novel genome-editing tools, and their activity has been greatly enhanced by eight additional mutations, thus named ABE8e. However, elevated catalytic activity was concomitant with frequent generation of bystander mutations. This bystander effect precludes its safe applications required in human gene therapy. To develop next-generation ABEs that are both catalytically efficient and positionally precise, we performed combinatorial engineering of NG-ABE8e. We identify a novel variant (NG-ABE9e), which harbors nine mutations. NG-ABE9e exhibits robust and precise base-editing activity in human cells, with more than 7-fold bystander editing reduction at some sites, compared with NG-ABE8e. To demonstrate its practical utility, we used NG-ABE9e to correct the frequent T17M mutation in Rhodopsin for autosomal dominant retinitis pigmentosa. It reduces bystander editing by ∼4-fold while maintaining comparable efficiency. NG-ABE9e possesses substantially higher activity than NG-ABEmax and significantly lower bystander editing than NG-ABE8e in rice. Therefore, this study provides a versatile and improved adenine base editor for genome editing.
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Registered trials
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.