Cystic Fibrosis: New Trends in Therapy Methods

Abstract

This review provides information on recent advancements in the treatment of cystic fi brosis and presents interim results from ongoing clinical trials. Various scientifi c databases, including Scopus, Web of Science, and EMBASE, were utilized during the preparation of this review.The results of studies on new drugs such as acebilustat, LAU-7b, JBT-101 designed for anti-infl ammatory therapy of this disease are also presented.The review describes various approaches to cystic fi brosis therapy — substance ABO401, SP-101. It includes clinical trial results for the adeno-associated vector 4D-710, liposomal nanoparticles, including the drugs MRT5005, RCT2100, the corrector galicaftor, as well as the drugs lumacaftor+ivacaftor, tezacaftor+ivacaftor, tezacaftor+ivacaftor+elexacaftor и tezacaftor+vanzacaftor+deutivacaftor.Special attention is given to transgene delivery using vectors with a detailed discussion of the advantages and disadvantages of this method. The main modern genome editing techniques, their capabilities, advantages and disadvantages are also described.The results of the study on the oligosaccharide structures, which reduces sputum viscosity in patients with cystic fi brosis, are presented. This reduction in viscosity enhances the effectiveness of the antibiotic aztreonam, which is active against the Burkholderia cepacia complex — a group of pathogens, which is often responsible for infl ammation in cystic fi brosis patients.The role of targeted therapy as a factor capable of signifi cantly reducing disease severity was highlighted. Targeted therapy drugs can partially restore the function of the abnormal protein in cystic fi brosis patients, thereby reducing symptom severity and signifi cantly improving the patient’s quality of life. The necessity of further development in this fi eld was emphasized.

Keywords

• cystic fibrosis
• review
• targeted therapy
• genetic therapy
• gene editing
• genetic vector