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CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease

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Huntington’s Disease

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1780))

Abstract

This chapter describes the potential use of viral-mediated gene transfer in the central nervous system for genome editing in the context of Huntington’s disease. Here, we provide protocols that cover the design of various genome editing strategies, the cloning of CRISPR/Cas9 elements into lentiviral vectors, and the assessment of cleavage efficiency, as well as potential unwanted effects.

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Acknowledgments

We would like to thank Maria Rey for the preparation of the figures.

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Authors and Affiliations

  1. Laboratory of Neurotherapies and Neuromodulation (LNCM), Neuroscience Research Center (CRN), Lausanne University Hospital (CHUV), Lausanne, Switzerland

    Gabriel Vachey & Nicole Déglon

Authors
  1. Gabriel Vachey
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  2. Nicole Déglon
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Corresponding author

Correspondence to Nicole Déglon .

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Editors and Affiliations

  1. Brain Repair Group, Cardiff University, Cardiff, United Kingdom

    Sophie V. Precious

  2. Brain Repair Group, Cardiff University, Cardiff, United Kingdom

    Anne E. Rosser

  3. Brain Repair Group, Cardiff University, Cardiff, United Kingdom

    Stephen B. Dunnett

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Vachey, G., Déglon, N. (2018). CRISPR/Cas9-Mediated Genome Editing for Huntington’s Disease. In: Precious, S., Rosser, A., Dunnett, S. (eds) Huntington’s Disease. Methods in Molecular Biology, vol 1780. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-7825-0_21

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  • DOI: https://doi.org/10.1007/978-1-4939-7825-0_21

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-7824-3

  • Online ISBN: 978-1-4939-7825-0

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