Targeted Therapy for Cystic Fibrosis in Children

A mutation in the CFTR gene causes the malfunctioning of the CFTR protein that is located on the apical membrane of epithelial cells and functions as a chloride channel. The discovery of CFTR modulators is an advance in cystic fibrosis treatment. Objective: To analyze modern Russian and foreign lite...

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Published inInnovacionnaâ medicina Kubani (Online) no. 4; pp. 142 - 147
Main Authors Burlutskaya, A. V., Brisin, V. Yu, Statova, A. V., Zenkina, O. Yu, Pisotskaya, J. V.
Format Journal Article
LanguageEnglish
Russian
Published Scientific Research Institute, Ochapovsky Regional Clinical Hospital no. 1 17.11.2023
Subjects
cftr modulators
children
genes
mutation
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Summary:A mutation in the CFTR gene causes the malfunctioning of the CFTR protein that is located on the apical membrane of epithelial cells and functions as a chloride channel. The discovery of CFTR modulators is an advance in cystic fibrosis treatment. Objective: To analyze modern Russian and foreign literature on targeted therapy for cystic fibrosis in children. We also describe the history of cystic fibrosis, evolution in its diagnosis and treatment, and mechanisms behind targeted therapy for cystic fibrosis.
ISSN:2541-9897
2541-9897
DOI:10.35401/2541-9897-2023-8-4-142-147