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Overview of Adeno-Associated Viral Vectors

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Gene Delivery to Mammalian Cells

Part of the book series: Methods in Molecular Biology™ ((MIMB,volume 246))

Abstract

The use of adeno-associated virus (AAV) as a gene transfer vector has been steadily increasing over the past several years. A AV vectors have been particularly useful for applications where sustained gene expression is required. Prolonged in vivo expression following A AV treatment has been seen in the liver (1,2), brain (3,4), skeletal muscle (5,6), lung (7,8), and hematopoietic stem cells (9,10) of animal models. Therapeutic benefit from A AV treatment has been shown in a number of preclinical models of disease, including animal models of coagulopathies (11,12), lysosomal storage diseases (13,14), vision defects (15,16), and amino acid disorders (17). Clinical trials using A AV for the treatment of hemophilia B have begun, and early reports from these trials have been promising (18). In this introductory chapter to AAV, we will provide a brief overview of the molecular biology of this virus, an overview of methods of vector production, and a brief summary of the use of alternate AAV serotypes. The following chapters will then focus on specific methods and techniques for AAV transduction of the organs listed previously.

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Authors and Affiliations

  1. Department of Pathology, University of Alabama at Birmingham, Birmingham, AL

    Thomas M. Daly

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  1. Thomas M. Daly
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Editors and Affiliations

  1. Bio-Rad Laboratories, Hercules, CA

    William C. Heiser

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© 2004 Humana Press Inc., Totowa, NJ

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Daly, T.M. (2004). Overview of Adeno-Associated Viral Vectors. In: Heiser, W.C. (eds) Gene Delivery to Mammalian Cells. Methods in Molecular Biology™, vol 246. Humana Press. https://doi.org/10.1385/1-59259-650-9:157

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  • DOI: https://doi.org/10.1385/1-59259-650-9:157

  • Publisher Name: Humana Press

  • Print ISBN: 978-1-58829-095-3

  • Online ISBN: 978-1-59259-650-8

  • eBook Packages: Springer Protocols

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