Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis.

  title={Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis.},
  author={Marjolein Mijnders and Bertrand Kleizen and Ineke Braakman},
  journal={Current opinion in pharmacology},
Pharmacological intervention to treat the lethal genetic disease cystic fibrosis has become reality, even for the severe, most common folding mutant F508del CFTR. CFTR defects range from absence of the protein, misfolding that leads to degradation rather than cell-surface localization (such as F508del), to functional chloride-channel defects on the cell surface. Corrector and potentiator drugs improve cell-surface location and channel activity, respectively, and combination therapy of two… CONTINUE READING
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Discovery and characterization of ABBV/GLPG-2222, a novel first generation CFTR corrector

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