Challenges and strategies for cystic fibrosis lung gene therapy.

  title={Challenges and strategies for cystic fibrosis lung gene therapy.},
  author={David R Koehler and Mary M. Hitt and Jim Hu},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  volume={4 2},
Gene replacement therapy represents an interesting new approach for the treatment of cystic fibrosis (CF) lung disease. Basic research suggests that CF gene therapy is feasible, but major technological challenges must be addressed before clinical applications are likely to succeed. Therapeutic genes can be delivered to and expressed in human airways, but… CONTINUE READING