Gene therapy in cystic fibrosis.

@article{Flotte2001GeneTI,
  title={Gene therapy in cystic fibrosis.},
  author={Terence R. Flotte and Beth L. Laube},
  journal={Chest},
  year={2001},
  volume={120 3 Suppl},
  pages={124S-131S}
}
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

Citations

Publications citing this paper.
Showing 1-10 of 32 extracted citations

References

Publications referenced by this paper.
Showing 1-10 of 56 references

A threshold regional dose of AAV vector is necessary to detect transduction in the lower airways of rhesus macaques [abstract

  • SE Beck, BL Laube, CT Barberena
  • Pediatr Pulmonol
  • 2000

Deposition of aerosolized AAV vectors in the lungs of rhesus macaques [abstract

  • SE Beck, BL Laube, R Adams
  • Pediatr Pulmonol
  • 1999

Gene therapy in pediatrics

  • TR Flotte, BJ Byrne
  • eds. Oski’s pediatrics. 3rd ed. Philadelphia,
  • 1999

Recent advances in gene transfer methods for the treatment of cystic fibrosis

  • TR Flotte
  • Current Research in Ion Channel Modulat
  • 1998

Similar Papers

Loading similar papers…