CFTR Modulator Therapy for Rare CFTR Mutants Article Swipe
Marco Mergiotti
,
Alessandra Murabito
,
Giulia Prono
,
Alessandra Ghigo
·
YOU?
·
· 2022
· Open Access
·
· DOI: https://doi.org/10.3390/jor2020005
YOU?
·
· 2022
· Open Access
·
· DOI: https://doi.org/10.3390/jor2020005
Cystic fibrosis (CF), the most common genetic disease among the Caucasian population, is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR), a chloride epithelial channel whose dysfunction results in severe airway obstruction and inflammation, eventually leading to respiratory failure. The discovery of the CFTR gene in 1989 provided new insights into the basic genetic defect of CF and allowed the study of potential therapies targeting the aberrant protein. In recent years, the approval of “CFTR modulators”, the first molecules designed to selectively target the underlying molecular defects caused by specific CF-causing mutations, marked the beginning of a new era in CF treatment. These drugs have been demonstrated to significantly improve lung function and ameliorate the quality of life of many patients, especially those bearing the most common CFTR mutatant F508del. However, a substantial portion of CF subjects, accounting for ~20% of the European CF population, carry rare CFTR mutations and are still not eligible for CFTR modulator therapy, partly due to our limited understanding of the molecular defects associated with these genetic alterations. Thus, the implementation of models to study the phenotype of these rare CFTR mutations and their response to currently approved drugs, as well as to compounds under research and clinical development, is of key importance. The purpose of this review is to summarize the current knowledge on the potential of CFTR modulators in rescuing the function of rare CF-causing CFTR variants, focusing on both investigational and clinically approved molecules.
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Metadata
- Type
- article
- Language
- en
- Landing Page
- https://doi.org/10.3390/jor2020005
- https://www.mdpi.com/2673-527X/2/2/5/pdf?version=1649155798
- OA Status
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- Cited By
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- References
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- Related Works
- 10
- OpenAlex ID
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All OpenAlex metadata
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https://openalex.org/W4226144357Canonical identifier for this work in OpenAlex
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https://doi.org/10.3390/jor2020005Digital Object Identifier
- Title
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CFTR Modulator Therapy for Rare CFTR MutantsWork title
- Type
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articleOpenAlex work type
- Language
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enPrimary language
- Publication year
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2022Year of publication
- Publication date
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2022-04-05Full publication date if available
- Authors
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Marco Mergiotti, Alessandra Murabito, Giulia Prono, Alessandra GhigoList of authors in order
- Landing page
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https://doi.org/10.3390/jor2020005Publisher landing page
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https://www.mdpi.com/2673-527X/2/2/5/pdf?version=1649155798Direct link to full text PDF
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goldOpen access status per OpenAlex
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https://www.mdpi.com/2673-527X/2/2/5/pdf?version=1649155798Direct OA link when available
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Cystic fibrosis, Cystic fibrosis transmembrane conductance regulator, Medicine, Chloride channel, Population, Mutation, Potentiator, Phenotype, Genetic enhancement, Bioinformatics, Genetic disorder, Biology, Disease, Gene, Pharmacology, Cancer research, Genetics, Internal medicine, Cell biology, Environmental healthTop concepts (fields/topics) attached by OpenAlex
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10Total citation count in OpenAlex
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2025: 1, 2024: 5, 2023: 2, 2022: 2Per-year citation counts (last 5 years)
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10Other works algorithmically related by OpenAlex
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