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

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.