medgenМедицинская генетикаMedical Genetics2073-7998Publishing House «Genius Media» LLC10.25557/2073-7998.2024.09.3-17medgen-2551Research ArticleНАУЧНЫЙ ОБЗОРREVIEWОбзор текущих исследований по разработке генной терапии на основе геномного редактирования для лечения муковисцидозаReview of current research on the development of gene therapy for cystic fibrosis using genome editing techniquesСмирнихинаС. А.SmirnikhinaS. A.

 Смирнихина Светлана Анатольевна

115522, г. Москва, ул. Москворечье, д. 1

Svetlana A. Smirnikhina

1, Moskvorechye st., Moscow, 115522

smirnikhinas@gmail.comФГБНУ Медико-генетический научный центр имени академика Н.П. БочковаРоссияResearch Centre for Medical GeneticsRussian Federation202404122024239317Copyright © Смирнихина С.А., 20242024Смирнихина С.А.Smirnikhina S.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.medgen-journal.ru/jour/article/view/2551

Муковисцидоз (МВ) – частое моногенное заболевание, возникающее в результате мутаций в гене CFTR. Патогенетическая терапия, несмотря на ее высокую эффективность, подходит не всем пациентам, является пожизненной и сопряжена с побочными эффектами. В связи с этим в мире ведутся масштабные исследования, направленные на разработку этиотропной терапии МВ, в частности, основанные на геномном редактировании. В обзоре рассмотрены некоторые недостатки существующей терапии, описаны методы геномного редактирования и проанализированы опубликованные на текущий момент данные по генной терапии МВ с использованием методов геномного редактирования.

Cystic fibrosis (CF) is a prevalent monogenic disease caused by mutations in the CFTR gene. Although pathogenetic therapy is highly effective, it is not suitable for all patients, requires lifelong treatment, and is associated with side effects. Consequently, extensive research is being conducted globally to develop etiotropic therapy for CF, particularly focusing on genome editing methods. This review explores the limitations of current therapy, outlines genome editing techniques, and evaluates all currently available data on gene therapy for CF utilizing genome editing techniques

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