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Article

Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus

1 May 2013

Abstract excerpt

Recombinant adeno-associated virus (rAAV) vectors are great tools for gene transfer due to their ability to mediate long-term gene expression. rAAVs have been used successfully as gene transfer vehicles in multiple animal models of CNS disorders, and several clinical trials are currently underway. rAAV vectors have been used at various stages of development with no apparent toxicity. There are multiple ways of...

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PMID: 23686825
DOI: 10.1002/9780471729259.mc14d05s29

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