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Article

Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors

10 Feb 2015

Abstract excerpt

We describe receptor-targeted adeno-associated viral (AAV) vectors that allow genetic modification of rare cell types ex vivo and in vivo while showing no detectable off-targeting. Displaying designed ankyrin repeat proteins (DARPins) on the viral capsid and carefully depleting DARPin-deficient particles, AAV vectors were made specific for Her2/neu, EpCAM or CD4. A single intravenous administration of vector...

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PMID: 25665714
DOI: 10.1038/ncomms7246

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