Bioengineered factor IX molecules with increased catalytic activity improve the therapeutic index of gene therapy vectors for hemophilia B.

@article{BrunettiPierri2009BioengineeredFI,
  title={Bioengineered factor IX molecules with increased catalytic activity improve the therapeutic index of gene therapy vectors for hemophilia B.},
  author={Nicola Brunetti-Pierri and Nathan C. Grove and Yu Zuo and Rachel Edwards and Donna Palmer and Vincenzo Cerullo and J Teruya and Philip Ng},
  journal={Human gene therapy},
  year={2009},
  volume={20 5},
  pages={479-85}
}
Although the desire to develop gene therapy for hemophilia B is high, safety remains a concern. Therefore, improving the therapeutic index of gene therapy vectors is an important goal. Thus, we evaluated the use of three bioengineered factor IX (FIX) variants with improved catalytic activity in the context of the helper-dependent adenoviral vector. The… CONTINUE READING