ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
BEON: A Functional Fluorescence Reporter for Quantification and Enrichment of Adenine Base-Editing Activity.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 67 citations in OpenAlex.
- In vivo base editing of Asgr1 reduces blood lipids in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Development and testing of a versatile genome editing application reporter (V-GEAR) system.Molecular therapy. Methods & clinical development · 2024Article
- The Wild-Type tRNA Adenosine Deaminase Enzyme TadA Is Capable of Sequence-Specific DNA Base Editing.Chembiochem : a European journal of chemical biology · 2023Article
- Enrichment strategies to enhance genome editing.Journal of biomedical science · 2023Review
- Liver-specific in vivo base editing of Angptl3 via AAV delivery efficiently lowers blood lipid levels in mice.Cell & bioscience · 2023Article
- A luciferase reporter mouse model to optimize in vivo gene editing validated by lipid nanoparticle delivery of adenine base editors.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping.Molecular therapy. Methods & clinical development · 2023Article
- In vivo adenine base editing reverts C282Y and improves iron metabolism in hemochromatosis mice.Nature communications · 2022Article
- Development of a universal antibiotic resistance screening reporter for improving efficiency of cytosine and adenine base editing.The Journal of biological chemistry · 2022Article
- Mutation-specific reporter for optimization and enrichment of prime editing.Nature communications · 2022Article
- Article
- BVES is a novel interactor of ANO5 and regulates myoblast differentiation.Cell & bioscience · 2021Article
- Efficient precise in vivo base editing in adult dystrophic mice.Nature communications · 2021Article
- A Cas9-mediated adenosine transient reporter enables enrichment of ABE-targeted cells.BMC biology · 2020Article
Corrections and comments
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Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
Adenine base editor (ABE) is a new generation of genome-editing technology through fusion of Cas9 nickase with an evolved E. coli TadA (TadA∗) and holds great promise as novel genome-editing therapeutics for treating genetic disorders. ABEs can directly convert A-T to G-C in specific genomic DNA targets without introducing double-strand breaks (DSBs). We recently showed that computer program-assisted analysis of Sanger sequencing traces can be used as a low-cost and rapid alternative of deep sequencing to assess base-editing outcomes. Here we developed a rapid fluorescence-based reporter assay (Base Editing ON [BEON]) to quantify ABE efficiency. The assay relies on the restoration of the downstream green fluorescent protein (GFP) in ABE-mediated editing of a stop codon located within the guide RNA (gRNA). We showed that this assay can be used to screen for effective ABE variants, characterize the protospacer adjacent motif (PAM) requirement of a novel NNG-targeting ABE based on ScCas9, and enrich for edited cells. Finally, we demonstrated that the reporter assay allowed us to assess the feasibility of ABE editing to correct point mutations associated with dysferlinopathy. Taken together, the BEON assay would facilitate and simplify the studies with ABEs.
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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.