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      Engineering adeno-associated viruses for clinical gene therapy.

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          Abstract

          Clinical gene therapy has been increasingly successful owing both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.

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          Author and article information

          Journal
          Nat Rev Genet
          Nature reviews. Genetics
          Springer Science and Business Media LLC
          1471-0064
          1471-0056
          Jul 2014
          : 15
          : 7
          Affiliations
          [1 ] Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA.
          [2 ] 1] Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720-3220, USA. [2] Department of Bioengineering, and the Department of Molecular and Cell Biology, University of California, Berkeley, California 94720-3220, USA.
          Article
          nrg3742 NIHMS676802
          10.1038/nrg3742
          4393649
          24840552
          213f2c2e-f7d4-4e01-8afb-4869bebdf6a0
          History

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