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. 2015 Sep 24;163(1):17.
doi: 10.1016/j.cell.2015.09.003.

A combination therapy for cystic fibrosis

Jeffrey L Brodsky  1 , Raymond A Frizzell  2
Affiliations

A combination therapy for cystic fibrosis

Jeffrey L Brodsky et al. Cell. .

Abstract

The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.

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The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.
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