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Adeno-Associated Virus (AAV) Vectors in Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 1996)
Loot Price: R2,895
Discovery Miles 28 950
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Adeno-Associated Virus (AAV) Vectors in Gene Therapy (Paperback, Softcover reprint of the original 1st ed. 1996)
Series: Current Topics in Microbiology and Immunology, 218
Expected to ship within 10 - 15 working days
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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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