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Key to Delivery: The (Epi-)genome Editing Vector Toolbox

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Epigenome Editing

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1767))

Abstract

Curing a genetic disease by repairing the underlying genetic defect is a fascinating concept that has been addressed so far by gene compensation therapy. For this, a functional copy of the gene in question together with elements controlling its expression is produced as a vector and introduced ex vivo into the patient’s own cells that subsequently are reinfused. Alternatively, vectors are administered directly in vivo. Although this strategy resulted in impressive therapeutic benefits for patients, the ultimate goal of gene therapy, i.e., a cure by repairing the actual genetic or epigenetic defect, remained an unresolved task. With the advent of designer DNA-binding domains, this goal is coming into reach. These domains are either combined with nucleases and used as molecular precision scissors for introducing DNA breaks at defined sites in the cell’s genome preparing for position-selective DNA repair, or they are used as programmable DNA-binding units for positioning epigenome-modifying domains to predefined target sequences. However, for reaching its full potential, these components need to be delivered into cells in an efficient and safe manner. Here, we summarize current viral and non-viral delivery approaches applicable for genome and epigenome editing and discuss their respective advantages and limitations.

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Author information

Authors and Affiliations

  1. Laboratory for Infection Biology & Gene Transfer, Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

    Sabrina Just & Hildegard Büning

  2. Laboratory for AAV Vector Development, Center for Molecular Medicine Cologne (CMMC), University of Cologne, Cologne, Germany

    Hildegard Büning

  3. German Center for Infection Research (DZIF), Partner Site Hannover-Braunschweig, Braunschweig, Germany

    Hildegard Büning

Authors
  1. Sabrina Just
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  2. Hildegard Büning
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Correspondence to Hildegard Büning .

Editor information

Editors and Affiliations

  1. Department of Biochemistry, Institute of Biochemistry and Technical Biochemistry, Stuttgart, Germany

    Albert Jeltsch

  2. Department of Pathology and Medical Biology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands

    Marianne G. Rots

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Just, S., Büning, H. (2018). Key to Delivery: The (Epi-)genome Editing Vector Toolbox. In: Jeltsch, A., Rots, M. (eds) Epigenome Editing. Methods in Molecular Biology, vol 1767. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-7774-1_7

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  • DOI: https://doi.org/10.1007/978-1-4939-7774-1_7

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-7773-4

  • Online ISBN: 978-1-4939-7774-1

  • eBook Packages: Springer Protocols

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