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Recombinant adeno-associated virus vectors.

Authors: M, Hallek; A, Girod; M, Braun-Falco; C M, Wendtner; C, Bogedain; M, Hörer;

Recombinant adeno-associated virus vectors.

Abstract

Recombinant adeno-associated virus (rAAV) is a promising vector for somatic gene therapy due to the ability to transduce terminally-differentiated and non-dividing cells, the lack of any apparent pathogenicity, a low immunogenicity, a relatively high stability of transgene expression, and the potential for targeted integration. Improved methods of rAAV packaging allow the generation of concentrated and highly purified rAAV for clinical trials. Preclinical studies with rAAV are currently in progress for the treatment of a variety of inherited monogenic defects, but also for acquired diseases like HIV infection and cancer. Because of the broad host range of wild type AAV, rAAV is able to transduce a variety of human tissues, preferentially those of epitheloid origin, with high efficiency and therefore may be used for various clinical applications. Whilst several issues including safety, tissue tropism and methods to achieve site-specific integration need further improvement, rAAV certainly has a sufficient number of advantages to be seriously considered as a gene therapy vector.

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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
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