Molekulyarnaya Meditsina (Molecular medicine)Molekulyarnaya Meditsina (Molecular medicine)Молекулярная медицина1728-29182499-9490Russkiy Vrach Publishing House68929010.29296/24999490-2025-01-02ReviewsОбзорыReview ArticleNeurodegenerative diseases as a target for genome editing: from preclinical studies to clinical practiceНейродегенеративные заболевания как объект редактирования генома: от доклинических исследований к клинической практикеhttps://orcid.org/0000-0002-1605-7859TereshchenkoSergey Y.ТерещенкоСергей Юрьевич
Russian Federation

Head of the Clinical Department of Somatic and Mental Health of Children, Doctor of Medical Sciences, Professor

руководитель клинического отделения соматического и психического здоровья детей, доктор медицинских наук, профессор

legise@mail.ruhttps://orcid.org/0000-0003-1133-4447PotupchikTatyana V.ПотупчикТатьяна Витальевна
Russian Federation

Associate Professor, Department of Pharmacology and Clinical Pharmacology with a Postgraduate Course, Candidate of Medical Sciences

доцент кафедры фармакологии и клинической фармакологии с курсом постдипломного образования, кандидат медицинских наук

potupchik_tatyana@mail.ruhttps://orcid.org/0000-0003-0665-7428EvertLydia S.ЭвертЛидия Семеновна
Russian Federation

Chief Researcher, Clinical Department of Somatic and Mental Health of Children, Professor, Department of General Professional Disciplines, Medical Institute, Doctor of Medical Sciences

главный научный сотрудник клинического отделения соматического и психического здоровья детей, профессор кафедры общепрофессиональных дисциплин, Медицинский институт, доктор медицинских наук

lidiya_evert@mail.ruhttps://orcid.org/0009-0006-2500-9687AlievaLeila G.АлиеваЛейла Габиловна
Russian Federation

6th year student

студентка VI курса

alievaoxx@mail.ruhttps://orcid.org/0009-0003-9023-1936KozyrinaYuliana E.КозыринаЮлиана Евгеньевна
Russian Federation

6th year student

студентка VI курса

iuliana.kozyrina@mail.ruhttps://orcid.org/0009-0009-3505-5523MaremkulovAmin A.МаремкуловАмин Анзорович
Russian Federation

6th year student

студент VI курса

headshoter1985@mail.ruFederal Research Center “Krasnoyarsk Scientific Center of the Siberian Branch of the Russian Academy of Sciences” – Separate Subdivision Scientific Research Institute of Medical Problems of the NorthФедеральный исследовательский центр «Красноярский научный центр Сибирского отделения Российской академии наук» – обособленное подразделение Научно-исследовательский институт медицинских проблем СевераFederal State Budgetary Educational Institution of Higher Education “Krasnoyarsk State Medical University named after Professor V.F. Voino-Yasenetsky” of the Ministry of Health of the Russian FederationФГБОУ ВО «Красноярский государственный медицинский университет им. профессора В.Ф. Войно-Ясенецкого» Министерства здравоохранения Российской Федерации, Российская ФедерацияKhakass State University named after N.F. Katanov of the Ministry of Science and Higher Education of the Russian FederationФГБОУ ВО «Хакасский государственный университет им. Н.Ф. Катанова» Минобрнауки РоссииFederal State Autonomous Educational Institution of Higher Education “Peoples’ Friendship University of Russia named after Patrice Lumumba”, Ministry of Science and Higher Education of the Russian FederationФедеральное государственное автономное образовательное учреждение высшего образования «Российский университет дружбы народов имени Патриса Лумумбы» Минобрнауки РоссииFederal State Autonomous Educational Institution of Higher Education “N.I. Pirogov Russian National Research Medical University” of the Ministry of Health of the Russian FederationФГАОУ ВО «Российский национальный исследовательский медицинский университет им. Н.И. Пирогова» Министерства здравоохранения Российской Федерации0803202523116261408202514082025Copyright ©; 2025, Russkiy Vrach Publishing HouseCopyright ©; 2025, ИД "Русский врач"2025Russkiy Vrach Publishing HouseИД "Русский врач"https://journals.eco-vector.com/1728-2918/article/view/689290

Objective. To analyze the current state of preclinical and clinical studies in genome editing for neurodegenerative diseases, evaluate its potential impact on clinical practice, and examine ethical aspects of these technologies’ application.

Material and methods. A systematic literature review was conducted for the period 2016-2024 using PubMed, Cochrane Library, ClinicalTrials.gov, SAGE Premier, Springer, and Wiley Journals databases, using key words: “genome editing”, “CRISPR”, “neurodegenerative diseases”, “clinical trials”, “ethics”.

Results. Key genetic targets for genome editing in Alzheimer’s disease (APP, PSEN1/2, APOE), Parkinson’s disease (LRRK2, PARK7, SNCA), and Huntington’s disease (HTT) are examined. Results of key preclinical studies demonstrating the effectiveness of various genome editing approaches are analyzed. The success of initial clinical trials of genome editing technologies in related fields and their significance for developing neurodegenerative disease therapies are discussed. Ethical aspects of genome editing application in the nervous system are considered.

Conclusion. Despite significant progress in preclinical studies, the transition to clinical application of genome editing technologies in neurodegenerative diseases requires addressing multiple technical, biological, and ethical challenges. Success in clinical trials in related fields provides a foundation for developing effective therapeutic strategies.

Цель исследования. Анализ текущего состояния доклинических и клинических исследований в области редактирования генома при нейродегенеративных заболеваниях, оценить потенциальное влияние на клиническую практику и рассмотреть этические аспекты применения данных технологий.

Материал и методы. Проведен систематический анализ литературы за период 2015–2024 гг. в базах данных PubMed, Cochrane Library, ClinicalTrials.gov, SAGE Premier, Springer и Wiley Journals с использованием ключевых слов: «редактирование генома», «CRISPR», «нейродегенеративные заболевания», «клинические испытания», «этика».

Результаты. Рассмотрены основные генетические мишени для редактирования генома при болезни Альцгеймера (APP, PSEN1/2, APOE), болезни Паркинсона (LRRK2, PARK7, SNCA) и хорее Хантингтона. Проанализированы результаты ключевых доклинических исследований, продемонстрировавших эффективность различных подходов к геномному редактированию. Обсуждены успехи первых клинических испытаний технологий редактирования генома в смежных областях и их значение для развития терапии нейродегенеративных заболеваний. Рассмотрены этические аспекты применения геномного редактирования в нервной системе.

Заключение. Несмотря на значительный прогресс в доклинических исследованиях, переход к клиническому применению технологий редактирования генома при нейродегенеративных заболеваниях требует решения ряда технических, биологических и этических проблем. Успехи клинических испытаний в смежных областях создают основу для разработки эффективных терапевтических стратегий.

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