Adeno-Associated Virus (AAV) Vectors in Gene Therapy

Author:   Kenneth I. Berns ,  Catherine Giraud
Publisher:   Springer-Verlag Berlin and Heidelberg GmbH & Co. KG
Edition:   Softcover reprint of the original 1st ed. 1996
Volume:   218
ISBN:  

9783642802096


Pages:   173
Publication Date:   28 January 2012
Format:   Paperback
Availability:   Manufactured on demand   Availability explained
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Adeno-Associated Virus (AAV) Vectors in Gene Therapy


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Overview

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).

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Author:   Kenneth I. Berns ,  Catherine Giraud
Publisher:   Springer-Verlag Berlin and Heidelberg GmbH & Co. KG
Imprint:   Springer-Verlag Berlin and Heidelberg GmbH & Co. K
Edition:   Softcover reprint of the original 1st ed. 1996
Volume:   218
Dimensions:   Width: 15.50cm , Height: 1.00cm , Length: 23.50cm
Weight:   0.295kg
ISBN:  

9783642802096


ISBN 10:   3642802095
Pages:   173
Publication Date:   28 January 2012
Audience:   Professional and scholarly ,  Professional & Vocational
Format:   Paperback
Publisher's Status:   Active
Availability:   Manufactured on demand   Availability explained
We will order this item for you from a manufactured on demand supplier.

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