Polyethylenimine shows properties of interest for cystic fibrosis gene therapy.

@article{Ferrari1999PolyethylenimineSP,
  title={Polyethylenimine shows properties of interest for cystic fibrosis gene therapy.},
  author={Stefano Ferrari and Andrea Pettenazzo and N Garbati and Franco Zacchello and J. P. Behr and Maurizio Scarpa},
  journal={Biochimica et biophysica acta},
  year={1999},
  volume={1447 2-3},
  pages={219-25}
}
Before being considered for a cystic fibrosis (CF) gene therapy trial, any gene delivery agent must be able to show that it produces low levels of toxicity as well as being able to protect the DNA from nuclease degradation. Here we show that complexes of linear polyethylenimine (L-PEI) and DNA can repeatedly be administered to animals (up to 21 consecutive days) without eliciting an immune response against PEI/DNA particles or inducing toxic side effects due to accumulation of PEI in the lungs… CONTINUE READING