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Designing Non-viral Targeted Integrating Vectors for Genome Engineering in Vertebrates

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Site-directed insertion of transgenes

Part of the book series: Topics in Current Genetics ((TCG,volume 23))

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Abstract

Genome engineering for biomedicine and biotechnology requires the integration of transgenes at specific sites in safe genomic environments. These specific integrations are needed to avoid position effects, insertional mutagenesis and chromosome abnormalities. Several efforts during the last decade led to the development of nonviral approaches based on DNA-modifying enzymes by exploiting the cellular mechanisms of cut-and-paste transposition and homologous recombination (HR). These methods include the use of zinc finger nucleases, meganucleases, site-specific recombinases such as ΦC31 integrase, Cre and Flp recombinases and transposase-based systems to achieve the integration of foreign DNA at a desired genomic position. Moreover, many teams are now investigating strategies to alter the site-specificity of these enzymes and precisely target safe insertion sites.

This review attempts to provide a general overview on recent advances in designing nonviral site-directed integrating vectors, required for both gene therapy and animal transgenesis. We discuss the advantages of such engineered vectors, the strategies employed to improve their targeting efficiency and consider their limitations in terms of safety and activity.

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Abbreviations

DSB:

Double strand break

ZF:

Zinc finger

gDNA:

Genomic DNA

HR:

Homologous recombination

ITR:

Inverted terminal repeat

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Authors and Affiliations

  1. Institute of Systems and Synthetic Biology, Genopole, CNRS, Université d’Evry Val d’Essonne, Evry, France

    Ludivine Sinzelle

  2. Institute of Systems and Synthetic Biology, Genopole, CNRS, Université d’Evry Val d’Essonne, Genavenir 3, Genopole campus 1, 1, rue Pierre Fontaine, 91058, Evry, France

    Nicolas Pollet

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  1. Ludivine Sinzelle
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  2. Nicolas Pollet
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Correspondence to Nicolas Pollet .

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  1. , UFR des Sciences Pharmaceutiques, Université François Rabelais, Parc Grandmont, Tours, 37200, France

    Sylvaine Renault

  2. Cellectis SA, Rue de la Croix Jarry 8, Paris, 75013, France

    Philippe Duchateau

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Sinzelle, L., Pollet, N. (2013). Designing Non-viral Targeted Integrating Vectors for Genome Engineering in Vertebrates. In: Renault, S., Duchateau, P. (eds) Site-directed insertion of transgenes. Topics in Current Genetics, vol 23. Springer, Dordrecht. https://doi.org/10.1007/978-94-007-4531-5_2

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