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A history of genome editing in mammals

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Abstract

Genome editing is now a routine procedure in many mammalian genetics laboratories. The ostensibly short but intense history of genome-editing approaches illustrates how a disruptive technology can universally colonize a field when this new methodology, conceived to alter mammalian genomes at specific locations, is found to efficiently and robustly deliver results. This review summarizes the early development of genome editing using nucleases, from the pioneering experiments using yeast meganucleases, to the latest prokaryotic nucleases used for precise genome manipulation. Gene-editing nucleases belong to one of three known categories: zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR) and their associated proteins (Cas) tools. All operate on the same principle; they are all capable of inducing a double-strand break (DSB) at a defined genomic sequence that is subsequently corrected by endogenous DNA repair mechanisms. DSBs can be repaired through non-homologous end joining (NHEJ), resulting in small insertions and/or deletions (INDELs) and, hence, often leading to gene disruption. Alternatively, DSBs can be repaired through homology-driven repair (HDR), in the presence of donor homologous DNA sequences, resulting in gene-editing events.

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Acknowledgements

LM´s laboratory is funded through the Spanish Ministry of Economy and Competitiveness (MINECO) [BIO2015-70978], the Biomedical and Biological Sciences (BMBS) European Cooperation in Science and Technology (COST) action [BM1308 SALAAM], and the Spanish Biomedical Research Networking Centre on Rare Diseases (CIBERER) from the National Institute of Heath Carlos III (Instituto de Salud Carlos III, ISCIII). The authors thank Kenneth McCreath for English language editing assistance.

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  1. Departamento de Biología Molecular y Celular, Centro Nacional de Biotecnología (CNB-CSIC), Campus de Cantoblanco, Darwin, 3, 28049, Madrid, Spain

    Almudena Fernández, Santiago Josa & Lluis Montoliu

  2. CIBERER, Instituto de Salud Carlos III, Madrid, Spain

    Almudena Fernández, Santiago Josa & Lluis Montoliu

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  1. Almudena Fernández
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  2. Santiago Josa
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  3. Lluis Montoliu
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Correspondence to Lluis Montoliu.

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Fernández, A., Josa, S. & Montoliu, L. A history of genome editing in mammals. Mamm Genome 28, 237–246 (2017). https://doi.org/10.1007/s00335-017-9699-2

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