Adenoviral Vector DNA for Accurate Genome Editing with Engineered Nucleases

Overview

Journal Nat Methods

Specialties Biomedical Engineering
Pathology

Date 2014 Aug 26

PMID 25152084

Citations 74

Authors

Maarten Holkers

Ignazio Maggio

Sara F D Henriques

Josephine M Janssen

Toni Cathomen

Manuel A F V Goncalves

Affiliations

Soon will be listed here.

Abstract

Engineered sequence-specific nucleases and donor DNA templates can be customized to edit mammalian genomes via the homologous recombination (HR) pathway. Here we report that the nature of the donor DNA greatly affects the specificity and accuracy of the editing process following site-specific genomic cleavage by transcription activator-like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-Cas9 nucleases. By applying these designer nucleases together with donor DNA delivered as protein-capped adenoviral vector (AdV), free-ended integrase-defective lentiviral vector or nonviral vector templates, we found that the vast majority of AdV-modified human cells underwent scarless homology-directed genome editing. In contrast, a significant proportion of cells exposed to free-ended or to covalently closed HR substrates were subjected to random and illegitimate recombination events. These findings are particularly relevant for genome engineering approaches aiming at high-fidelity genetic modification of human cells.

Citing Articles

Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs.

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Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy.

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Applications of Innovation Technologies for Personalized Cancer Medicine: Stem Cells and Gene-Editing Tools.

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High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells.

Tasca F, Brescia M, Liu J, Janssen J, Mamchaoui K, Goncalves M Mol Ther Nucleic Acids. 2023; 31:746-762.

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Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants.

Wang Q, Liu J, Janssen J, Goncalves M Nucleic Acids Res. 2023; 51(7):3465-3484.

PMID: 36928106 PMC: 10123109. DOI: 10.1093/nar/gkad165.

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