Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells, and has attracted a significant amount of attention in the field, especially in clinical-stage experimental therapeutic strategies. The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for various therapeutic applications has thus far proven to be one of the safest strategies for gene therapies. This review will provide an overview of some important factors to consider in the use of AAV as a vector for gene therapy. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png BioDrugs Springer Journals

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

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Publisher
Springer International Publishing
Copyright
Copyright © 2017 by The Author(s)
Subject
Biomedicine; Molecular Medicine; Antibodies; Cancer Research; Pharmacotherapy
ISSN
1173-8804
eISSN
1179-190X
D.O.I.
10.1007/s40259-017-0234-5
Publisher site
See Article on Publisher Site

Abstract

There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells, and has attracted a significant amount of attention in the field, especially in clinical-stage experimental therapeutic strategies. The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for various therapeutic applications has thus far proven to be one of the safest strategies for gene therapies. This review will provide an overview of some important factors to consider in the use of AAV as a vector for gene therapy.

Journal

BioDrugsSpringer Journals

Published: Jul 1, 2017

References

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