ArticleMolecular therapy. Nucleic acids2019
Improving Editing Efficiency for the Sequences with NGH PAM Using xCas9-Derived Base Editors.
Article in Molecular therapy. Nucleic acids, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.
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Who cites it
7 citing papers in PubMed.
- Next-generation CRISPR technology for genome, epigenome and mitochondrial editing.Transgenic research · 2024Review
- Navigating the CRISPR/Cas Landscape for Enhanced Diagnosis and Treatment of Wilson's Disease.Cells · 2024Review
- Advances in CRISPR/Cas gene therapy for inborn errors of immunity.Frontiers in immunology · 2023Review
- PAM-flexible dual base editor-mediated random mutagenesis and self-activation strategies to improve CRISPRa potency.Molecular therapy. Methods & clinical development · 2022Article
- Use of hiPSC to explicate genomic predisposition to anthracycline-induced cardiotoxicity.Pharmacogenomics · 2021Review
- Recent advances of genome editing and related technologies in China.Gene therapy · 2020Review
- CRISPR-based strategies for targeted transgene knock-in and gene correction.Faculty reviews · 2020Review
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Authors and funding
9 authors.
Funding
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Abstract
The development of CRISPR/Cas9-mediated base editors (BEs) provided a versatile tool for precise genome editing. The recently developed xCas9-derived base editors (xBEs) that recognize the NG PAM substantially expand the targeting scope in the genome, while their editing efficiency needs to be improved. Here, we described an improved version of xBEs by fusing the BPNLS and Gam to the N terminus of xBEs (BPNLS-Gam-xBE3 and BPNLS-xABE), and this version of base editor displayed higher targeting efficiency for the majority of detected sites. By using this improved version of xBEs, we successfully created and corrected pathogenic mutations at genomic sites with the NGN protospacer-adjacent motif in human cells. Lastly, we used BPNLS-Gam-xBE3 to model pathogenic mutations in discarded human tripronuclear (3PN) zygotes, and no obvious off-targets and indels were detected. Taken together, the data in our study offer an efficient tool for precise genome editing and, thus, an enriched base editing toolkit.
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