Author:
Lomunova Maria A.,Gershovich Pavel M.
Abstract
Gene replacement therapies are novel therapeutic approaches that seek to tackle hereditary diseases caused by a congenital deficiency in a particular gene, when a functional copy of a gene can be delivered to the cells and tissues using various delivery systems. To do this, viral particles carrying a functional copy of the gene of interest and various nonviral gene delivery systems, including liposomes, nanoparticles, etc., can be used. In this review, we discuss the state of current knowledge regarding the molecular mechanisms and types of genetic mutations that lead to cystic fibrosis and highlight recent developments in gene therapy that can be leveraged to correct these mutations and to restore the physiological function of the carrier protein transporting sodium and chlorine ions in the airway epithelial cells. Restoration of carrier protein expression could lead to the normalization of ion and water transport across the membrane and induce a decrease in the viscosity of airway surface fluid, which is one of the pathological manifestations of this disease. This review also summarizes recently published preclinical and clinical data for various gene therapies to allow one to make some conclusions about future prospects for gene therapy in cystic fibrosis treatment.
Reference78 articles.
1. Classification of cystic fibrosis and related disorders. Report of a Joint Working Group of WHO/ICF (M)A/ECFS/ECFTN. World Health Organization. // J. Cyst. Fibros. 2002. V. 1. P. 5–8.
2. Orlov A.V., Simonova O.I., Roslavtseva E.A., Shadrin D.I. Cystic Fibrosis (clinical aspects, diagnostics, treatment, rehabilitation, clinical examination): Textbook for doctors. St. Petersburg: North-Western State Medical University. I.I. Mechnikov, 2014. 160 p.
3. Baranov A.A., Namazova-Baranova L.S., Simonova O.I., Kashirskaya N.Yu., Roslavtseva E.A., Gorinova Yu.V., Krasovskiy S.A., Selimzyanova L.R. // Pediatric pharmacology. 2015. V. 12. №5. P. 589–604.
4. Clinical recommendations. Cystic Fibrosis: microbiological diagnosis of chronic respiratory infection. Ministry of Health of the, 2020.
5. Yan Z., McCray P.B., Engelhardt J.F. // Hum. Mol. Genet. 2019. V. 28. № R1. P. R88–R94.
Cited by
3 articles.
订阅此论文施引文献
订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献