Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose.

@article{Wu2008OptimizationOS,
  title={Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose.},
  author={Zhijian Wu and Junjiang Sun and Tai-ping Zhang and Chaoying Yin and F. L. Yin and Terry Van Dyke and Richard Jude Samulski and Paul E Monahan},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2008},
  volume={16 2},
  pages={280-9}
}
Self-complementary adeno-associated virus (scAAV) vectors can significantly minimize the vector load required to achieve sustained transgene expression. In this study, transcriptional regulatory elements were systematically screened to produce constitutive and liver-specific scAAV factor IX (FIX) expression cassettes. In addition, optimization of GC content, cis- regulatory elements, and codon usage in the human FIX (hFIX) transgene increased expression 4-20-fold. A vector was developed that… CONTINUE READING

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