Recent Strategic Advances in CFTR Drug Discovery: An Overview

Marco Rusnati, Pasqualina D'Ursi, Nicoletta Pedemonte, Chiara Urbinati, Robert C Ford, Elena Cichero, Matteo Uggeri, Alessandro Orro, Paola Fossa

Research output: Contribution to journalReview articlepeer-review


Cystic fibrosis transmembrane conductance regulator (CFTR)-rescuing drugs have already transformed cystic fibrosis (CF) from a fatal disease to a treatable chronic condition. However, new-generation drugs able to bind CFTR with higher specificity/affinity and to exert stronger therapeutic benefits and fewer side effects are still awaited. Computational methods and biosensors have become indispensable tools in the process of drug discovery for many important human pathologies. Instead, they have been used only piecemeal in CF so far, calling for their appropriate integration with well-tried CF biochemical and cell-based models to speed up the discovery of new CFTR-rescuing drugs. This review will give an overview of the available structures and computational models of CFTR and of the biosensors, biochemical and cell-based assays already used in CF-oriented studies. It will also give the reader some insights about how to integrate these tools as to improve the efficiency of the drug discovery process targeted to CFTR.

Original languageEnglish
JournalInt J Mol Sci
Issue number7
Publication statusPublished - Mar 31 2020


  • Biosensing Techniques
  • Computational Biology
  • Cystic Fibrosis/drug therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator/chemistry
  • Drug Discovery/methods
  • Humans
  • Models, Molecular
  • Protein Conformation


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