avkАрхивъ внутренней медициныThe Russian Archives of Internal Medicine2226-67042411-6564“SINAPS” LLC10.20514/2226-6704-2025-15-4-275-283NFCRYUavk-2045Research ArticleОБЗОРНЫЕ СТАТЬИREVIEW ARTICLESМуковисцидоз: новые тенденции в методах терапииCystic Fibrosis: New Trends in Therapy Methodshttps://orcid.org/0009-0008-1783-7612СучковаП. А.SuchkovaP. A.

Сучкова Полина Александровна — студент 6-го курса педиатрического факультета 

Санкт-Петербург

Polina A. Suchkova — 6th year student of the pediatric faculty 

Saint-Petersburg

ponty.stop@mail.ruhttps://orcid.org/0009-0007-8475-0915ПановаС. А.PanovaS. A.

Панова София Алексеевна — студент 6-го курса педиатрического факультета

Санкт-Петербург

Sofia A. Panova — 6th year student of the pediatric faculty 

Saint-Petersburg

sof.anov@gmal.comhttps://orcid.org/0009-0005-2754-0108ЛисенкоО. Я.LisenkoO. Ya.

Лисенко Оксана Яковлевна — студент 6-го курса педиатрического факультета

Санкт-Петербург

Oksana Ya. Lisenko — 6th year student of the pediatric faculty

Saint-Petersburg

oksanalisenko01@mail.ruhttps://orcid.org/0000-0002-9939-3443РаевскийК. П.RaevskijK. P.

Раевский Кирилл Павлович — аспирант кафедры терапии факультета фундаментальной медицины

Москва

Kirill P. Raevsky — postgraduate student of the Department of Therapy, Faculty of Fundamental Medicine

Moscow

raevskiikp@my.msu.ruФГБОУ ВО Санкт-Петербургский государственный педиатрический медицинский университетРоссияSaint-Petersburg State Pediatric Medical UniversityRussian FederationФГБОУ ВО Медицинский научно-образовательный институт, Московский государственный университет им. М.В. ЛомоносоваРоссияMoscow Research and Education Institute of the Lomonosov Moscow State UniversityRussian Federation202529072025154275283Copyright © Сучкова П.А., Панова С.А., Лисенко О.Я., Раевский К.П., 20252025Сучкова П.А., Панова С.А., Лисенко О.Я., Раевский К.П.Suchkova P.A., Panova S.A., Lisenko O.Y., Raevskij K.P.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.medarhive.ru/jour/article/view/2045

Несмотря на увеличение продолжительности и заметное улучшение качества жизни больных, на сегодняшний день муковисцидоз остается одним из тяжелых пульмонологических заболеваний, ведущим к инвалидизации. В связи с актуальностью проблемы терапии данного заболевания, ежегодно стартуют разработки новых препаратов для лечения муковисцидоза.

Данный обзор описывает новейшие достижения в лечении муковисцидоза, также представлены промежуточные результаты ведущихся клинических исследований. В процессе подготовки обзора были использованы различные базы научных данных: Scopus, Web of Science, EMBASE.

Описаны результаты исследований новых препаратов, предназначенных для противовоспалительной терапии данного заболевания — ацебилустата, препарата LAU-7b, JBT-101.

Рассмотрены результаты исследования альгината олигосахарида, снижающего вязкость мокроты у больных муковисцидозом. Эффект препарата был продемонстрирован на примере усиления действия антибиотика азтреонама, эффективного против Burkholderia cepacia complex — группы патогенных микроорганизмов, часто поражающих дыхательную систему больных муковисцидозом.

Описаны исследования различных препаратов генной терапии муковисцидоза — вещества ABO401, препарата SP-101, представлены результаты клинических исследований аденоассоциированного вектора 4D-710, липосомных наночастиц, в том числе препаратов MRT5005, RCT2100, таргетной терапии корректора галикафтора, комбинаций ивакафтор+лумакафтор, тезакафтор+ивакафтор, ивакафтор+тезакафтор +элексакафтор и ванзакафтор+тезакафтор+деутивакафтор.

Особенное внимание в обзоре было уделено доставке трансгена при помощи векторов, описаны преимущества и недостатки данного метода. Описаны основные современные методы геномного редактирования, их возможности, преимущества и недостатки.

Показана роль таргетной терапии как фактора, способного значительно уменьшать тяжесть течения заболевания. Препараты таргетной терапии способны частично восстанавливать функцию аномального белка у больных муковисцидозом, а значит снижать степень проявления симптомов и значительно повышать качество жизни пациента. Описана необходимость дальнейшей разработки данного направления.

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.

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The authors declare that there are no conflicts of interest present.