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Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina

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Adeno-Associated Virus Vectors

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1950))

Abstract

Degenerative retinal diseases such as retinitis pigmentosa (RP) and Leber’s congenital amaurosis (LCA) may lead to blindness without effective treatment. With the rapid advancement of the CRISPR/Cas9 genome editing technology, in vivo application of CRISPR/Cas9 holds immense potential for treatment of these diseases. Adeno-associated virus (AAV) vectors are an ideal gene transfer tool for delivery of CRISPR components to the retina. Here, we describe a protocol for utilizing an AAV-based CRISPR/Cas9 system for in vivo genome editing in the retina.

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Authors and Affiliations

  1. Ocular Gene Therapy Core, National Eye Institute, NIH, Bethesda, MD, USA

    Wenhan Yu & Zhijian Wu

Authors
  1. Wenhan Yu
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  2. Zhijian Wu
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Corresponding author

Correspondence to Zhijian Wu .

Editor information

Editors and Affiliations

  1. Department of Neurosciences, University of California, San Diego, La Jolla, CA, USA

    Michael J. Castle

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Yu, W., Wu, Z. (2019). Use of AAV Vectors for CRISPR-Mediated In Vivo Genome Editing in the Retina. In: Castle, M. (eds) Adeno-Associated Virus Vectors. Methods in Molecular Biology, vol 1950. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9139-6_7

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  • DOI: https://doi.org/10.1007/978-1-4939-9139-6_7

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  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-9138-9

  • Online ISBN: 978-1-4939-9139-6

  • eBook Packages: Springer Protocols

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