Creation and Research of Cell Models of Hereditary Neurodegenerative Diseases Using Directed Genome Editing

• Published in: Neurochemical journal 2021-10, Vol.15 (4), p.353-358
• Main Authors: Medvedev, S. P., Malankhanova, T. B., Valetdinova, K. R., Zakian, S. M.
• Format: Article
• Language: English
• Subjects: Biomedical and Life Sciences; Biomedicine; Neurosciences; Review Articles
• ISSN: 1819-7124; 1819-7132
• DOI: 10.1134/S1819712421040073

—Neurodegenerative diseases make up a large part of overall morbidity around the world. In particular, an increase in the average age of the population in most developed countries leads to a drastic increase in the proportion of patients with diagnoses such as Alzheimer’s disease and Parkinson’s disease. The existing methods of treatment are predominantly symptomatic and, in some cases can have a pronounced clinical effect, however, they cannot prevent the continuing death of neurons or reverse neurodegenerative process. To a large extent, these problems are associated with an insufficient understanding of the molecular genetic mechanisms that underlie pathogenesis, as well as with the lack of models that allow qualitative and quantitative data on the processes occurring in the neurons of patients to be obtained. The development of technologies of induced pluripotency, directed differentiation of pluripotent cells, and gene editing using programmed nucleases, makes it possible to significantly expand the arsenal of available research tools, especially in the search for new targets for drug and gene therapy.