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Publication details

Document type
Journal articles

Document subtype
Full paper

Title
Discovery of CFTR modulators for the treatment of cystic fibrosis

Participants in the publication
Miquéias Lopes-Pacheco (Author)
BioISI - Biosystems & Integrative Sciences Institute
 Dep. Química e Bioquímica
 BioISI
 Nicoletta Pedemonte (Author)
Guido Veit (Author)

Summary
Introduction: Cystic fibrosis (CF) is a life-threatening inherited disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel expressed at the apical membrane of secretory epithelia. CF leads to multiorgan dysfunction with progressive deterioration of lung function being the major cause of untimely death. Conventional CF therapies target only symptoms and consequences downstream of the primary genetic defect and the current life expectancy and quality of life of these individuals are still very limited.\n\nArea covered: CFTR modulator drugs are novel-specialized therapies that enhance or even restore functional expression of CFTR mutants and have been approved for clinical use for individuals with specific CF genotypes. This review summarizes classical approaches used for the pre-clinical development of CFTR correctors and potentiators as well as emerging strategies aiming to accelerate modulator development and expand theratyping efforts.\n\nExpert opinion: Highly effective CFTR modulator drugs are expected to deeply modify the disease course for the majority of individuals with CF. A multitude of experimental approaches have been established to accelerate the development of novel modulators. CF patient-derived specimens are valuable cell models to predict therapeutic effectiveness of existing (and novel) modulators in a precision medicine approach.

Date of Submisson/Request
2020-11-30
Date of Acceptance
2021-03-31
Date of Publication
2021-04-13

Where published
Expert Opinion on Drug Discovery

Publication Identifiers
ISSN - 1746-0441

Publisher
Informa UK Limited

Number of pages
17
Starting page
1
Last page
17

Document Identifiers
DOI - https://doi.org/10.1080/17460441.2021.1912732
URL - http://dx.doi.org/10.1080/17460441.2021.1912732

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APA
Miquéias Lopes-Pacheco, Nicoletta Pedemonte, Guido Veit, (2021). Discovery of CFTR modulators for the treatment of cystic fibrosis. Expert Opinion on Drug Discovery, 1-17. ISSN 1746-0441. eISSN . http://dx.doi.org/10.1080/17460441.2021.1912732

IEEE
Miquéias Lopes-Pacheco, Nicoletta Pedemonte, Guido Veit, "Discovery of CFTR modulators for the treatment of cystic fibrosis" in Expert Opinion on Drug Discovery, pp. 1-17, 2021. 10.1080/17460441.2021.1912732

BIBTEX
@article{52984, author = {Miquéias Lopes-Pacheco and Nicoletta Pedemonte and Guido Veit}, title = {Discovery of CFTR modulators for the treatment of cystic fibrosis}, journal = {Expert Opinion on Drug Discovery}, year = 2021, pages = {1-17}, }