Modern pathogenesis-based methods and development of new gene and cell-based methods for cystic fibrosis treatment

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Abstract

Cystic fibrosis is a monogenic autosomal recessive disorder caused by mutations in CFTR gene. Until recent days, cystic fibrosis therapy was limited to symptomatic treatment of respiratory infections and malabsorption. In last years pathogenetic therapy of the disease received significant progress and premises for development of new methods of gene therapy came into sight. In the review, modern methods of cystic fibrosis treatment are considered, some of them are already used in the clinic (pathogenesis-based therapy with CFTR modulators), while the other part is only developing (gene therapy, including genome editing and cell therapy).

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About the authors

S. A Smirnikhina

Research Centre for Medical Genetics

Email: smirnikhinas@gmail.com

A. V Lavrov

Research Centre for Medical Genetics; N.I. Pirogov Russian National Research Medical University

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