Novel opportunities for CFTR-targeting drug development using organoids

@inproceedings{Dekkers2013NovelOF,
  title={Novel opportunities for CFTR-targeting drug development using organoids},
  author={Johanna F. Dekkers and Cornelis K van der Ent and Jeffrey Matthijn Beekman},
  booktitle={Rare diseases},
  year={2013}
}
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR mutations lead to production of non-functional CFTR, reduced amounts of normal functioning CFTR or misfolded CFTR with defects in trafficking or function. For decades, CF treatment has been focused on the symptoms of CF, but pharmacotherapy using small molecules that target the basic defect of CF, the mutant CFTR protein, is now possible for a limited amount of subjects with… CONTINUE READING

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