Genome editing in neurodegenerative diseases: risk analysis, technological challenges, and prospects for clinical application
- Autores: Evert L.S.1,2, Potupchik T.V.3, Veselova O.F.3, Svetlakova I.S.4, Panchenko V.A.4, Kuzhasheva R.R.5, Melnikova A.N.6
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Afiliações:
- Federal Research Center “Krasnoyarsk Scientific Center of the Siberian Branch of the Russian Academy of Sciences”
- Khakass State University named after N.F. Katanov of the Ministry of Science and Higher Education of the Russian Federation
- 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
- Federal State Autonomous Educational Institution of Higher Education “Russian National Research Medical University named after N.I. Pirogov” of the Ministry of Health of the Russian Federation
- Federal State Budgetary Educational Institution of Higher Education “Bashkir State Medical University” of the Ministry of Health of the Russian Federation
- Regional Budgetary Healthcare Institution “Zolotukhinskaya Central District Hospital”
- Edição: Volume 23, Nº 2 (2025)
- Páginas: 21-31
- Seção: Reviews
- URL: https://journal-vniispk.ru/1728-2918/article/view/293270
- DOI: https://doi.org/10.29296/24999490-2025-02-03
- ID: 293270
Citar
Resumo
Objective. To analyze the potential risks and technological limitations of using genome editing methods in neurodegenerative diseases, as well as to assess the prospects for their implementation in clinical practice.
Material and methods. A systematic analysis of the literature for the period 2014–2024 in the databases PubMed, Cochrane Library, ClinicalTrials.gov, SAGE Premier, Springer and Wiley Journals. The key risks of using genome editing technologies are considered, including inappropriate effects, immunological reactions, and long-term consequences of changes in the DNA of nervous tissue.
Results. The main technological limitations are analyzed, including problems of delivery across the blood-brain barrier, low editing efficiency in postmitotic neurons, and the complexity of long-term expression of components of editing systems. The prospects of introducing technologies into clinical practice are assessed, taking into account the current regulatory landscape in various countries.
Conclusion. Despite significant technological challenges and potential risks, the development of genome editing techniques opens up prospects for the creation of effective treatments for neurodegenerative diseases. Key areas of further research include improving the safety and specificity of editing, optimizing delivery systems, and developing methods for long-term monitoring of the effects of genetic modifications in the nervous system.
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##article.viewOnOriginalSite##Sobre autores
Lydia Evert
Federal Research Center “Krasnoyarsk Scientific Center of the Siberian Branch of the Russian Academy of Sciences”; Khakass State University named after N.F. Katanov of the Ministry of Science and Higher Education of the Russian Federation
Autor responsável pela correspondência
Email: lidiya_evert@mail.ru
ORCID ID: 0000-0003-0665-7428
Scientific Research Institute of Medical Problems of the North, Chief Researcher, Clinical Department of Somatic and Mental Health of Children, Medical Institute, Doctor of Medical Sciences
Rússia, Partizan Zheleznyak str., 3g, Krasnoyarsk, 660022; Lenin Ave., 90, Abakan, 655017Tatyana 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
Rússia, Partizan Zheleznyak str., 1, Krasnoyarsk, 660022Olga Veselova
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: Veselovaof@mail.ru
ORCID ID: 0000-0002-6126-665X
Head of the Department of Pharmacology and Clinical Pharmacology with a postgraduate course, Candidate of Medical Sciences
Rússia, Partizan Zheleznyak str., 1, Krasnoyarsk, 660022Irina Svetlakova
Federal State Autonomous Educational Institution of Higher Education “Russian National Research Medical University named after N.I. Pirogov” of the Ministry of Health of the Russian Federation
Email: irinasvetlakova2000@gmail.ru
ORCID ID: 0009-0005-7622-2827
6th year student
Rússia, Ostrovityanova str., 1, Moscow, 117997Viktor Panchenko
Federal State Autonomous Educational Institution of Higher Education “Russian National Research Medical University named after N.I. Pirogov” of the Ministry of Health of the Russian Federation
Email: viktor-panchenko@inbox.ru
ORCID ID: 0009-0001-2122-8501
6th year student
Rússia, Ostrovityanova str., 1, Moscow, 117997Rina Kuzhasheva
Federal State Budgetary Educational Institution of Higher Education “Bashkir State Medical University” of the Ministry of Health of the Russian Federation
Email: rkuzhasheva@bk.ru
ORCID ID: 0009-0001-7884-4209
6th year student
Rússia, Lenin str., 3, Ufa, 450008, Republic of Bashkortostan;Anastasia Melnikova
Regional Budgetary Healthcare Institution “Zolotukhinskaya Central District Hospital”
Email: nasmel00@mail.ru
ORCID ID: 0009-0006-6084-2578
doctor of the emergency department
Rússia, Kirova str., 81, Zolotukhino work settlement, Zolotukhinsky district, Kursk region, 306020Bibliografia
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