GENE THERAPY: CURRENT ACHIEVEMENTS AND FUTURE PERSPECTIVES

• Maratova Lobar

  Gulistan state university 12-24 group 2nd year student maratovalobar176@gmail.com

  Author

Gene therapy represents one of the most promising innovations in modern molecular medicine. It aims to treat or even cure genetic and acquired diseases by correcting or replacing defective genes within human cells. Over the past decade, advances in genome editing tools, viral and non-viral vector systems, and personalized medicine have brought gene therapy closer to clinical reality. This paper examines current developments, successful applications, and the challenges that must be overcome for its widespread use in the future.

Ginn, S. L., Amaya, A. K., Alexander, I. E., Edelstein, M., & Abedi, M. R. (2018). Gene therapy clinical trials worldwide to 2017: An update. The Journal of Gene Medicine, 20(5), e3015.

Naldini, L. (2019). Genetic engineering of hematopoietic stem cells: Current status and future perspectives. Nature Reviews Genetics, 20(4), 247–262.

Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018). Gene therapy comes of age. Science, 359(6372), eaan4672.

Li, H., Yang, Y., Hong, W., Huang, M., Wu, M., & Zhao, X. (2020). Applications of genome editing technology in the targeted therapy of human diseases: Mechanisms, advances and prospects. Signal Transduction and Targeted Therapy, 5(1), 1–23.

Smith, D., & Collins, J. (2021). CRISPR-Cas9: A powerful tool for genome engineering and therapeutic potential. Trends in Biotechnology, 39(8), 882–898.

High, K. A., & Roncarolo, M. G. (2019). Gene therapy. The New England Journal of Medicine, 381(5), 455–464.

Doudna, J. A., & Charpentier, E. (2020). The new frontier of genome editing with CRISPR-Cas9. Science, 367(6480), eaax9181.

Musunuru, K. (2022). Moving from discovery to therapy: The path for gene editing in cardiovascular disease. Circulation Research, 130(2), 223–241.

Kaiser, J. (2021). FDA approves first in vivo CRISPR gene editing therapy. Science, 374(6566), 925–926.

Orkin, S. H., & Reilly, P. (2023). Ethical and regulatory considerations in gene therapy and genome editing. Nature Medicine, 29(1), 10–19.

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