Effect of VX-770 in Persons with Cystic Fibrosis and the G551D- CFTR Mutation
Claudia L. Ordoñez, Melissa A. Ashlock, Steven D. Freedman, Scott D. Sagel, Michael W. Konstan, Steven M. Rowe, J.P. Clancy, Lynn M. Rose, Peter R. Durie, Douglas B. Hornick, Bonnie W. Ramsey, Ahmet Uluer, Frank J. Accurso, Michael Boyle, Scott H. Donaldson, Moira L. Aitken, Qunming Dong, Nicole Mayer‐Hamblett, Anne J. Stone, Jiuhong Zha, Eric R. Olson, Preston W. Campbell, Jordan Dunitz, Richard B. Moss, Ronald C. Rubenstein, Joseph M. Pilewski
Abstract
A new approach in the treatment of cystic fibrosis involves improving the function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro.
Topics & Concepts
Cystic fibrosisMutationMedicineInternal medicineGeneticsBiologyGeneCystic Fibrosis Research Advances