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Discov Med. 2010 May;9(48):399-403.

Reengineered AAV vectors: old dog, new tricks.

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Gene Therapy Center, The University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.


Adeno-associated viral (AAV) vectors have emerged in recent years as powerful tools for therapeutic gene transfer. Successes in clinical trials and the discovery of several hundreds of naturally occurring AAV isolates have triggered efforts to understand and manipulate this deceptively simple parvovirus for a myriad of gene therapy applications. Exciting breakthroughs based on directed evolution of novel tissue-specific variants from combinatorial AAV libraries have been reported. Recent approaches driven by the availability of structural information have yielded a new generation of reengineered AAV vectors.

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