Recombinant adeno-associated virus vectors for gene therapy.

  title={Recombinant adeno-associated virus vectors for gene therapy.},
  author={Thomas J. Conlon and Terence R. Flotte},
  journal={Expert opinion on biological therapy},
  volume={4 7},
Recombinant adeno-associated virus (rAAV) vectors are based on a non-pathogenic human parvovirus (AAV) that is unique in its ability to persist in human cells without causing any pathologic effects. Studies of the potential barriers to rAAV-mediated transduction of relatively resistant cells has led to an understanding of the mechanisms of cell attachment and entry, cytoplasmic translocation, nuclear entry, conversion to active double-stranded DNA, activation of transcription and establishment… CONTINUE READING

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