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Human Hepatocyte Transduction with Adeno-Associated Virus Vector

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Hepatocytes

Part of the book series: Methods in Molecular Biology ((MIMB,volume 2544))

Abstract

As the adeno-associated virus (AAV) vectors hold unique advantages over other viral vectors, AAV gene therapy has accumulated rapid progress and development. Liver-targeted gene therapy by AAV vectors has been successfully applied in clinical trials for many diseases. Low transduction efficiency and high prevalence of neutralizing antibodies (Nabs), however, are the major obstacles to further translate this therapeutic strategy into clinical trials. Pre-clinical evaluation on hepatocytes could help to elucidate the tropism of AAV serotypes for liver-targeted gene therapy, and could also provide a test model to develop novel AAV mutants with Nabs evasion and high liver tropism. Here, we described the basic laboratory procedure to apply the AAV vector to transduce human hepatocytes in vitro and in vivo with some tips gained from our own experience.

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Author information

Author notes

  1. Zhenwei Song and Wenwei Shao have been equally contributed to this chapter.

Authors and Affiliations

  1. Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

    Zhenwei Song, Liujiang Song, Xieolei Pei & Chengwen Li

  2. Academy of Medical Engineering and Translational Medicine, Tianjin University, Tianjin, China

    Wenwei Shao

Authors
  1. Zhenwei Song
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  2. Wenwei Shao
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  3. Liujiang Song
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  4. Xieolei Pei
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  5. Chengwen Li
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Corresponding author

Correspondence to Chengwen Li .

Editor information

Editors and Affiliations

  1. Research Institute for Frontier Medicine, Sapporo Medical University, School of Medicine, Sapporo, Hokkaido, Japan

    Naoki Tanimizu

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© 2022 The Author(s), under exclusive license to Springer Science+Business Media, LLC, part of Springer Nature

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Song, Z., Shao, W., Song, L., Pei, X., Li, C. (2022). Human Hepatocyte Transduction with Adeno-Associated Virus Vector. In: Tanimizu, N. (eds) Hepatocytes. Methods in Molecular Biology, vol 2544. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-2557-6_5

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  • DOI: https://doi.org/10.1007/978-1-0716-2557-6_5

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  • Publisher Name: Humana, New York, NY

  • Print ISBN: 978-1-0716-2556-9

  • Online ISBN: 978-1-0716-2557-6

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