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Hepatic Delivery of Artificial Micro RNAs Using Helper-Dependent Adenoviral Vectors

  • Protocol
SiRNA Delivery Methods

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1364))

Abstract

The potential of RNA interference (RNAi)-based gene therapy has been demonstrated in many studies. However, clinical application of this technology has been hampered by a paucity of efficient and safe methods of delivering the RNAi activators. Prolonged transgene expression and improved safety of helper-dependent adenoviral vectors (HD AdVs) makes them well suited to delivery of engineered artificial intermediates of the RNAi pathway. Also, AdVs’ natural hepatotropism makes them potentially useful for liver-targeted gene delivery. HD AdVs may be used for efficient delivery of cassettes encoding short hairpin RNAs and artificial primary microRNAs to the mouse liver. Methods for the characterization of HD AdV-mediated delivery of hepatitis B virus-targeting RNAi activators are described here.

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Acknowledgments

The authors’ laboratory receives financial assistance from the South African National Research Foundation (NRF, GUNs 81768, 81692, 68339, 85981 & 77954), Medical Research Council and Poliomyelitis Research Foundation.

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Authors and Affiliations

  1. Wits/SAMRC Antiviral Gene Therapy Research Unit, School of Pathology, Health Sciences Faculty, University of the Witwatersrand, Private Bag 3, Johannesburg, WITS 2050, South Africa

    Carol Crowther, Betty Mowa & Patrick Arbuthnot

Authors
  1. Carol Crowther
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  2. Betty Mowa
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  3. Patrick Arbuthnot
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Corresponding author

Correspondence to Patrick Arbuthnot .

Editor information

Editors and Affiliations

  1. Beckman Research Institute of City of Hope, Duarte, California, USA

    Kato Shum

  2. Beckman Research Institute of City of Hope, Duarte, California, USA

    John Rossi

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Crowther, C., Mowa, B., Arbuthnot, P. (2016). Hepatic Delivery of Artificial Micro RNAs Using Helper-Dependent Adenoviral Vectors. In: Shum, K., Rossi, J. (eds) SiRNA Delivery Methods. Methods in Molecular Biology, vol 1364. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3112-5_20

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  • DOI: https://doi.org/10.1007/978-1-4939-3112-5_20

  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-3111-8

  • Online ISBN: 978-1-4939-3112-5

  • eBook Packages: Springer Protocols

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