Targeted Therapy for Cystic Fibrosis in Children

Abstract

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.

Keywords

• cftr modulators
• children
• genes
• mutation