Article
In vivo nasal potential difference: techniques and protocols for assessing efficacy of gene transfer in cystic fibrosis.
Human gene therapy - 1 Apr 1995
Knowles M R, Paradiso A M, Boucher R C
Abstract excerpt
Cystic fibrosis (CF) is a monogenetic disease that is associated with chronic airways disease and early death. The pulmonary disease reflects mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, and associated abnormal epithelial ion transport, including defective cAMP-mediated (CFTR) Cl- secretion and an accelerated rate of basal Na+ transport. With the development of vectors for...
Topics
- Adolescent
- Adult
- Aged
- Aged, 80 and over
- Amiloride
- Biological Transport
- Calcium
- Child
- Child, Preschool
- Chlorides
- Cyclic AMP
- Cystic Fibrosis