Gene therapy in cystic fibrosis.

  title={Gene therapy in cystic fibrosis.},
  author={Terence R. Flotte and Beth L. Laube},
  volume={120 3 Suppl},
Theoretically, cystic fibrosis transmembrane conductance regulator (CFTR) gene replacement during the neonatal period can decrease morbidity and mortality from cystic fibrosis (CF). In vivo gene transfers have been accomplished in CF patients. Choice of vector, mode of delivery to airways, translocation of genetic information, and sufficient expression level of the normalized CFTR gene are issues that currently are being addressed in the field. The advantages and limitations of viral vectors… CONTINUE READING


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