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Review
. 2021 Apr 23;10(5):486.
doi: 10.3390/antibiotics10050486.

Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review

Rosa María Girón Moreno  1 , Marta García-Clemente  2 , Layla Diab-Cáceres  3 , Adrián Martínez-Vergara  4 , Miguel Ángel Martínez-García  5 , Rosa Mar Gómez-Punter  1
Affiliations
Review

Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review

Rosa María Girón Moreno et al. Antibiotics (Basel). .

Abstract

Cystic fibrosis (CF) is a genetic disease that causes absence or dysfunction of a protein named transmembrane conductance regulatory protein (CFTR) that works as an anion channel. As a result, the secretions of the organs where CFTR is expressed are very viscous, so their functionality is altered. The main cause of morbidity is due to the involvement of the respiratory system as a result of recurrent respiratory infections by different pathogens. In recent decades, survival has been increasing, rising by around age 50. This is due to the monitoring of patients in multidisciplinary units, early diagnosis with neonatal screening, and advances in treatments. In this chapter, we will approach the different therapies used in CF for the treatment of symptoms, obstruction, inflammation, and infection. Moreover, we will discuss specific and personalized treatments to correct the defective gene and repair the altered protein CFTR. The obstacle for personalized CF treatment is to predict the drug response of patients due to genetic complexity and heterogeneity of uncommon mutations.

Keywords: CFTR modulator; RNA therapy; antibiotic; cystic fibrosis; editing gene; genetic therapy; inflammation; obstruction; treatment.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

Figure 1
Figure 1
Oscillating devices.
Figure 2
Figure 2
Algorithm for treating PA first isolate.
Figure 3
Figure 3
CFTR modulators. 1: transcription; 2: translation; 3: posttranslational modification; 4: protein trafficking; 5: surface expression of functional CFTR; 6: CFTR turnover. CFTR: cystic fibrosis transmembrane conductance regulator. NMD nonsense-mediated mRNA decay. The Roman numerals (I–VII) represent the different classes of CFTR mutations and the place where they originate, and therefore also the target of treatment. Adapted from Cuyx, De Boeck. Treating the Underlying CFTR defect in Patients with CF [168].
Figure 4
Figure 4
Other therapies.
See this image and copyright information in PMC

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