References

1. 1. Anzalone A.V., Gao X.D., Podracky C.J. et al. Programmable deletion, replacement, integration and inversion of large DNA sequences with twin prime editing // Nat Biotechnol. 2022. V. 40. P. 731-740. https://doi.org/10.1038/s41587-021-01133-w
2. 2. Arjmand B., Larijani B., Hosseini M.S. et al. The horizon of gene therapy in modern medicine: advances and challenges // Adv. Exp. Med. Biol. 2020. V. 1247. P. 33-64. https://doi.org/10.1007/5584_2019_463
3. 3. Balon K., Sheriff A., Jacków J. et al. Targeting cancer with CRISPR/Cas9-based therapy // Int. J. Mol. Sci. 2022. V. 23. Art. 573. https://doi.org/10.3390/ijms23010573
4. 4. Birney E. A society-wide conversation is needed about germline genome editing using CRISPR // Nat. Med. 2024. V. 30. P 30-32. https://doi.org/10.1038/s41591-023-02681-1
5. 5. De Graeff N., De Proost L., Munsie M. ‘Ceci n’est pas un embryon?’ The ethics of human embryo model research // Nat. Methods. 2023. V. 20. P. 1863-1867. https://doi.org/10.1038/s41592-023-02066-9
6. 6. Doudna J.A., Charpentier E. Genome editing. The new frontier of genome engineering with CRISPR-Cas9 // Science. 2014. V. 346 (6213). Art. 1258096. https://doi.org/10.1126/science.1258096
7. 7. Doudna J.A., Sternberg S.H. A Crack in Creation: Gene Editing and the Unthinkable Power to Control Evolution. San Francisco: Mariner, 2017. FDA (Federal Drug Association). Casgevy approval. 2024. URL: https://www.fda.gov/vaccines-blood-biologics/casgevy
8. 8. Foreman A.L., Liddell K., Franklin S. et al. Human embryo models: the importance of national policy and governance review // Curr. Op. Genetics Develop. 2023. V. 82. Art. 102103. https://doi.org/10.1016/j.gde.2023.102103
9. 9. Goguen R.P., Chen M.J., Dunkley O.R.S. Gene therapy to cure HIV infection // Virologie (Montrouge). 2023. V. 27. P. 63-84. https://doi.org/10.1684/vir.2023.1024
10. 10. Grebenshchikova E.G., Andreyuk D.S., Vasiliev A.V. et al.Russia’s stance on gene-edited humans // Nature. 2019. V. 575. P. 596. http://dx.doi.org/10.1038/ d41586-019-03617-x
11. 11. Griciuc A., Federico A. N., Natasan J. et al. Gene therapy for Alzheimer’s disease targeting CD33 reduces amyloid beta accumulation and neuroinflammation // Hum. Mol. Genet. 2020. V. 29. P. 2920-2935. https://doi.org/10.1093/hmg/ddaa179
12. 12. Guo C., Ma X., Gao F. et al. Off-target effects in CRISPR/ Cas9 gene editing // Front. Bioeng. Biotechnol. 2023. V. 11. Art. 1143157. https://doi.org/10.3389/fbioe.2023.1143157
13. 13. Harari Y.N. Homo deus: A Brief History of Tomorrow. New York: Penguin, 2017.
14. 14. Hsu P.D., Scott D.A., Weinstein J.A. et al. DNA targeting specificity of RNA-guided Cas9 nucleases // Nat. biotechnol. 2013. V. 31. P. 827-832. https://doi.org/10.1038/nbt.2647
15. 15. Huxley A. Brave New World. London: Nobel, 2024.
16. 16. ISSCR Guidelines. ISSCR Guidelines for Stem Cell Research and Clinical Translation. https://www.isscr.org/docs/default-source/all-iss-cr-guidelines/2021-guidelines/isscr-guidelines-for-stem-cell-research-and-clinical-translation-2021.pd-f?sfvrsn=979d58b1_4
17. 17. Kim A., Lalonde K., Truesdell A. et al. New avenues for the treatment of Huntington’s disease // Int. J. Mol. Sci. 2021. V. 22. Art. 8363. https://doi.org/10.3390/ijms22168363
18. 18. Kondkar A.A., Abu-Amero K.K. Leber congenital amaurosis: Current genetic basis, scope for genetic testing and personalized medicine. Exp Eye Res. 2019. V. 189. Art. 107834. https://doi.org/10.1016/j.exer.2019.107834
19. 19. Ledford H. CRISPR gene editing in human embryos wreaks chromosomal mayhem // Nature. 2020. V. 583. P. 17-18.
20. 20. Liu G., Lin Q., Jin S. et al. The CRISPR-Cas toolbox and gene editing technologies // Molecular Cell. 2022. V. 82. № 2. P. 333-347. https://doi.org/10.1016/j.molcel.2021.12.002
21. 21. Matthews K.R.W., Iltis A.S., Marquez N.G. et al. Rethinking human embryo research policies // Hastings Center Rep. 2021. 2021. V. 51. P. 47-51. https://doi.org/10.1002/hast.1215
22. 22. Pierce E.A., Aleman T.S., Jayasundera K.T. et al. Gene editing for CEP290-associated retinal degeneration // New Engl. J. Med. 2024. V. 390. P. 1972-1984. https://doi.org/10.1056/NEJMoa2309915
23. 23. Rivron N.C., Arias A.M., Pera M.F. et al. An ethical framework for human embryology with embryo models // Cell. 2023. V. 186. P. 3548-3557. https://doi.org/10.1016/j.cell.2023.07.028
24. 24. Sassin W., Donskikh O., Gnes A. et al. Evolutionary environments. Homo sapiens - an endangered species? Innsbruck: Studia Universitätsverlag, 2018.
25. 25. Scholefield J., Harrison P.T. Prime editing-an update on the field // Gene Therapy. 2021. V. 28. P. 396-401. https://doi.org/10.1038/s41434-021-00263-9
26. 26. Statement from the Organising Committee of the Third International Summit on Human Genome Editing, Royal Society. https://royalsociety.org/news/2023/03/statement-third-international-summit-human-genome-editing
27. 27. Subbaraman N. Limit on lab-grown human embryos dropped by stem-cell body // Nature. 2021. V. 594. P. 18-19. https://doi.org/10.1038/d41586-021-01423-y
28. 28. Thompson A.A., Walters M.C., Kwiatkowski J. et al. Gene therapy in patients with transfusion-dependent β-thalassemia // New Engl. J. Med. 2018. V. 378. P. 1479-1493. https://doi.org/10.1056/NEJMoa1705342
29. 29. Warnock M. Report of the committee of inquiry into human ertilization and embryology. London: Her Majesty’s Stationery Office, 1984. http://www.hfea.gov.uk/docs/Warnock_Report_of_the_Committee_of_Inquiry_into_Human_Fertilisation_and_Embryology_1984.pdf
30. 30. Zhao Z., Shang P., Mohanraju P. et al. Prime editing: advances and therapeutic applications // Trends Biotechnol. 2023. V. 41. P. 1000-1012.