Adeno-Associated Virus (AAV) Vectors in Gene Therapy

  • Kenneth I. Berns
  • Catherine Giraud

Part of the Current Topics in Microbiology and Immunology book series (CT MICROBIOLOGY, volume 218)

Table of contents

  1. Front Matter
    Pages I-IX
  2. K. I. Berns, C. Giraud
    Pages 1-23
  3. J. A. Chiorini, S. M. Wiener, L. Yang, R. H. Smith, B. Safer, N. P. Kilcoin et al.
    Pages 25-33
  4. K. K. Wong Jr., S. Chatterjee
    Pages 145-170
  5. Back Matter
    Pages 171-177

About this book

Introduction

Human gene therapy holds great promise for the cure of many genetic diseases. In order to achieve such a cure there are two requirements. First, the affected gene must be cloned, its se­ quence determined and its regulation adequately characterized. Second, a suitable vector for the delivery of a good copy of the affected gene must be available. For a vector to be of use several attributes are highly desirable: these include ability to carry the intact gene (although this may be either the genomic or the cDNA form) in a stable form, ability to introduce the gene into the desired cell type, ability to express the introduced gene in an appropriately regulated manner for an extended period of time, and a lack of toxicity for the recipient. Also of concern is the frequency of cell transformation and, in some cases, the ability to introduce the gene into nondividing stem cells. Sev­ eral animal viruses have been tested as potential vectors, but none has proven to have all the desired properties described above. For example, retroviruses are difficult to propagate in sufficient titers, do not integrate into nondividing cells, and are of concern because of their oncogenic properties in some hosts and because they integrate at many sites in the genome and, thus, are potentially insertional mutagens. Additionally, genes introduced by retroviral vectors are frequently expressed for relatively short periods of time. A second virus used as a vector in model systems has been adenovirus (Ad).

Keywords

chromosome gene gene therapy hematopoietic stem cell stem cells virus

Editors and affiliations

  • Kenneth I. Berns
    • 1
  • Catherine Giraud
    • 1
  1. 1.Hearst Microbiology Research Center, Department of MicrobiologyCornell University Medical CollegeNew YorkUSA

Bibliographic information

  • DOI https://doi.org/10.1007/978-3-642-80207-2
  • Copyright Information Springer-Verlag Berlin Heidelberg 1996
  • Publisher Name Springer, Berlin, Heidelberg
  • eBook Packages Springer Book Archive
  • Print ISBN 978-3-642-80209-6
  • Online ISBN 978-3-642-80207-2
  • Series Print ISSN 0070-217X
  • About this book