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

  title={Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors.},
  author={Robert C. M{\"u}nch and Anke Muth and Alexander Muik and Thorsten Friedel and Julia Schmatz and Birgit Dreier and Alexandra Trkola and Andreas Pl{\"u}ckthun and Hildegard Buening and Christian J Buchholz},
  journal={Nature communications},
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 targeted to the tumour antigen Her2/neu was sufficient to track 75% of all tumour sites… CONTINUE READING
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