Neurodegenerative diseases as a target for genome editing: from preclinical studies to clinical practice

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  • Authors: Tereshchenko S.Y.1, Potupchik T.V.2, Evert L.S.1,3, Alieva L.G.4, Kozyrina Y.E.5, Maremkulov A.A.5
  • Affiliations:
    1. Federal 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 North
    2. Federal 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 Federation
    3. Khakass State University named after N.F. Katanov of the Ministry of Science and Higher Education of the Russian Federation
    4. Federal 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 Federation
    5. Federal State Autonomous Educational Institution of Higher Education “N.I. Pirogov Russian National Research Medical University” of the Ministry of Health of the Russian Federation
  • Issue: Vol 23, No 1 (2025)
  • Pages: 16-26
  • Section: Reviews
  • URL: https://journals.eco-vector.com/1728-2918/article/view/689290
  • DOI: https://doi.org/10.29296/24999490-2025-01-02
  • ID: 689290

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Abstract

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.

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About the authors

Sergey Y. Tereshchenko

Federal 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 North

Author for correspondence.
Email: legise@mail.ru
ORCID iD: 0000-0002-1605-7859

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

Russian Federation, Partizan Zheleznyak str., 3G, Krasnoyarsk, 660022

Tatyana V. Potupchik

Federal 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 Federation

Email: potupchik_tatyana@mail.ru
ORCID iD: 0000-0003-1133-4447

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

Russian Federation, Partizan Zheleznyak str., 1, Krasnoyarsk, 660022

Lydia S. Evert

Federal 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 North; Khakass State University named after N.F. Katanov of the Ministry of Science and Higher Education of the Russian Federation

Email: lidiya_evert@mail.ru
ORCID iD: 0000-0003-0665-7428

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

Russian Federation, Partizan Zheleznyak str., 3G, Krasnoyarsk, 660022; Lenin Ave., 90, Abakan, 655017

Leila G. Alieva

Federal 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 Federation

Email: alievaoxx@mail.ru
ORCID iD: 0009-0006-2500-9687

6th year student

Russian Federation, Miklukho-Maklaya str., 6, Moscow, 117198

Yuliana E. Kozyrina

Federal State Autonomous Educational Institution of Higher Education “N.I. Pirogov Russian National Research Medical University” of the Ministry of Health of the Russian Federation

Email: iuliana.kozyrina@mail.ru
ORCID iD: 0009-0003-9023-1936

6th year student

Russian Federation, Ostrovityanova str., 1, Moscow, 117513

Amin A. Maremkulov

Federal State Autonomous Educational Institution of Higher Education “N.I. Pirogov Russian National Research Medical University” of the Ministry of Health of the Russian Federation

Email: headshoter1985@mail.ru
ORCID iD: 0009-0009-3505-5523

6th year student

Russian Federation, Ostrovityanova str., 1, Moscow, 117513

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