Open AccessA Combination Therapy for Cystic Fibrosis
Author(s) -
Jeffrey L. Brodsky,
Raymond A. Frizzell
Publication year - 2015
Publication title -
cell
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 26.304
H-Index - 776
eISSN - 1097-4172
pISSN - 0092-8674
DOI - 10.1016/j.cell.2015.09.003
Subject(s) - potentiator , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , biology , mutation , regulator , genetics , medicine , gene
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. To view this installment of Bench to Bedside, open or download the PDF
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