Genome editing in neurodegenerative diseases: risk analysis, technological challenges, and prospects for clinical application

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.