Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle.

@article{Salva2007DesignOT,
  title={Design of tissue-specific regulatory cassettes for high-level rAAV-mediated expression in skeletal and cardiac muscle.},
  author={Maja Z Salva and Charis L. Himeda and Phillip W L Tai and Eiko Nishiuchi and Paul Gregorevic and James M. Allen and Eric E. Finn and Quynh G Nguyen and Michael J Blankinship and Leonard A Meuse and Jeffrey S. Chamberlain and Stephen D. Hauschka},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2007},
  volume={15 2},
  pages={320-9}
}
Systemic delivery of recombinant adeno-associated virus (rAAV) 6 vectors mediates efficient transduction of the entire striated musculature, making this an attractive strategy for muscle gene therapy. However, owing to widespread transduction of non-muscle tissues, optimization of this method would benefit from the use of muscle-specific promoters. Most such promoters either lack high-level expression in certain muscle types or are too large for inclusion in rAAV vectors encoding… CONTINUE READING