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 liter...

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Main Authors: A. V. Burlutskaya, V. Yu. Brisin, A. V. Statova, O. Yu. Zenkina, J. V. Pisotskaya
Format: Article
Language:Russian
Published: Scientific Research Institute, Ochapovsky Regional Clinical Hospital no. 1 2023-11-01
Series:Инновационная медицина Кубани
Subjects:
Online Access:https://www.innovmedkub.ru/jour/article/view/773
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author A. V. Burlutskaya
V. Yu. Brisin
A. V. Statova
O. Yu. Zenkina
J. V. Pisotskaya
author_facet A. V. Burlutskaya
V. Yu. Brisin
A. V. Statova
O. Yu. Zenkina
J. V. Pisotskaya
author_sort A. V. Burlutskaya
collection DOAJ
description 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.
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publishDate 2023-11-01
publisher Scientific Research Institute, Ochapovsky Regional Clinical Hospital no. 1
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spelling doaj.art-aafe4a42e7454c35a491b8f0d2d020f62025-02-28T14:50:43ZrusScientific Research Institute, Ochapovsky Regional Clinical Hospital no. 1Инновационная медицина Кубани2541-98972023-11-010414214710.35401/2541-9897-2023-8-4-142-147420Targeted Therapy for Cystic Fibrosis in ChildrenA. V. Burlutskaya0V. Yu. Brisin1A. V. Statova2O. Yu. Zenkina3J. V. Pisotskaya4Kuban State Medical UniversityChildren’s Regional Clinical HospitalKuban State Medical UniversityKuban State Medical UniversityKuban State Medical UniversityA 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.https://www.innovmedkub.ru/jour/article/view/773cftr modulatorschildrengenesmutation
spellingShingle A. V. Burlutskaya
V. Yu. Brisin
A. V. Statova
O. Yu. Zenkina
J. V. Pisotskaya
Targeted Therapy for Cystic Fibrosis in Children
Инновационная медицина Кубани
cftr modulators
children
genes
mutation
title Targeted Therapy for Cystic Fibrosis in Children
title_full Targeted Therapy for Cystic Fibrosis in Children
title_fullStr Targeted Therapy for Cystic Fibrosis in Children
title_full_unstemmed Targeted Therapy for Cystic Fibrosis in Children
title_short Targeted Therapy for Cystic Fibrosis in Children
title_sort targeted therapy for cystic fibrosis in children
topic cftr modulators
children
genes
mutation
url https://www.innovmedkub.ru/jour/article/view/773
work_keys_str_mv AT avburlutskaya targetedtherapyforcysticfibrosisinchildren
AT vyubrisin targetedtherapyforcysticfibrosisinchildren
AT avstatova targetedtherapyforcysticfibrosisinchildren
AT oyuzenkina targetedtherapyforcysticfibrosisinchildren
AT jvpisotskaya targetedtherapyforcysticfibrosisinchildren