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Adeno-Associated Virus pp 93–118Cite as

Exploiting Natural Diversity of AAV for the Design of Vectors with Novel Properties

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 807))

Abstract

Twelve AAV serotypes have been described so far in human and nonhuman primate (NHP) populations while surprisingly high diversity of AAV sequences is detected in tissue biopsies. The analysis of these novel AAV sequences has indicated a rapid evolution of the viral genome both by accumulation of mutations and recombination. This chapter describes how this rich resource of naturally evolved sequences is used to derive gene transfer vectors with a wide array of activities depending on the nature of the cap gene used in the packaging system. AAV2-based recombinant genomes have been packaged in dozens of different capsid types, resulting in a wide array of “pseudotyped vectors” that constitute a rich resource for the development of gene therapy clinical trials. We describe a polymerase chain reaction-based molecular rescue method for novel AAV isolation that uses primers designed to recognize the highly conserved regions in known AAV isolates and generate amplicons across the hypervariable regions of novel AAV genomes present in the analyzed sample.

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Author information

Authors and Affiliations

  1. Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA

    Guangping Gao & Li Zhong

  2. INSERM U781, Hôpital Necker Enfants Malades and Universite Paris Descartes, Paris, France

    Olivier Danos

  3. University College London Cancer Institute, London, UK

    Olivier Danos

Authors
  1. Guangping Gao
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  2. Li Zhong
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  3. Olivier Danos
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Corresponding author

Correspondence to Guangping Gao .

Editor information

Editors and Affiliations

  1. Center of Excellence for Regenerative, Health Biotechnology (CERHB), University of Florida, Innovation Drive 13706, Alachua, 32615, Florida, USA

    Richard O. Snyder

  2. INSERM U649, Labo. Thérapie Génique, CHU Hôtel Dieu, bd. Jean Monnet 30, Nantes CX 1, 44035, France

    Philippe Moullier

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Gao, G., Zhong, L., Danos, O. (2012). Exploiting Natural Diversity of AAV for the Design of Vectors with Novel Properties. In: Snyder, R., Moullier, P. (eds) Adeno-Associated Virus. Methods in Molecular Biology, vol 807. Humana Press. https://doi.org/10.1007/978-1-61779-370-7_4

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  • DOI: https://doi.org/10.1007/978-1-61779-370-7_4

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  • Publisher Name: Humana Press

  • Print ISBN: 978-1-61779-369-1

  • Online ISBN: 978-1-61779-370-7

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