Current Topics in Microbiology and Immunology

Adeno-Associated Virus (AAV) Vectors in Gene Therapy

Editors: Berns, Kenneth I., Giraud, Catherine (Eds.)

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About this book

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).

Table of contents (9 chapters)

  • Biology of Adeno-associated Virus

    Berns, K. I. (et al.)

    Pages 1-23

  • The Roles of AAV Rep Proteins in Gene Expression and Targeted Integration

    Chiorini, J. A. (et al.)

    Pages 25-33

  • Packaging Systems for Adeno-associated Virus Vectors

    Trempe, J. P.

    Pages 35-50

  • The Challenges of Recombinant Adeno-associated Virus Manufacturing: Alternative Use of Adeno-associated Virus Plasmid/Liposome Complexes for Gene Therapy Applications

    Lebkowski, J. S. (et al.)

    Pages 51-59

  • Adeno-associated Virus Vectors for Gene Therapy of the Hematopoietic System

    Chatterjee, S. (et al.)

    Pages 61-73

Buy this book

eBook $109.00
price for USA (gross)
  • ISBN 978-3-642-80207-2
  • Digitally watermarked, DRM-free
  • Included format: PDF
  • ebooks can be used on all reading devices
  • Immediate eBook download after purchase
Softcover $149.00
price for USA
  • ISBN 978-3-642-80209-6
  • Free shipping for individuals worldwide
  • Usually dispatched within 3 to 5 business days.
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Bibliographic Information

Bibliographic Information
Book Title
Adeno-Associated Virus (AAV) Vectors in Gene Therapy
Editors
  • Kenneth I. Berns
  • Catherine Giraud
Series Title
Current Topics in Microbiology and Immunology
Series Volume
218
Copyright
1996
Publisher
Springer-Verlag Berlin Heidelberg
Copyright Holder
Springer-Verlag Berlin Heidelberg
eBook ISBN
978-3-642-80207-2
DOI
10.1007/978-3-642-80207-2
Softcover ISBN
978-3-642-80209-6
Series ISSN
0070-217X
Edition Number
1
Number of Pages
IX, 173
Number of Illustrations and Tables
22 b/w illustrations
Topics