Abstract
Annotatsiya: Ushbu maqolada gen terapiyasi tushunchasi, uning rivojlanish tarixi, asosiy mexanizmlari va amaliy qo‘llanilish sohalari keng yoritiladi. Irsiy kasalliklarni davolash imkoniyatlari, zamonaviy CRISPR/Cas9 texnologiyasining ahamiyati, gen transferi usullari, shuningdek, kelgusidagi istiqbollar haqida ilmiy tahlil olib boriladi. Shuningdek, gen terapiyasi bilan bog‘liq etik va huquqiy masalalar, xavfsizlik muammolari hamda personalizatsiyalashgan tibbiyotdagi o‘rni ham ko‘rib chiqiladi.
References
[1]: Collins F.S., Venter J.C. (2003). The Human Genome Project: Lessons from Large-Scale Biology. Science, 300(5617), 286–290.
[2]: Naldini L. (2015). Gene therapy returns to centre stage. Nature, 526(7573), 351–360.
[3]: Ginn S.L., Amaya A.K., Alexander I.E. (2018). Gene therapy clinical trials worldwide to 2017: An update. The Journal of Gene Medicine, 20(5), e3015.
[4]: Blaese R.M. et al. (1995). T lymphocyte-directed gene therapy for ADA- SCID: Initial trial results after 4 years. Science, 270(5235), 475–480.
[5]: Hacein-Bey-Abina S. et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science, 302(5644), 415–419.
[6]: Kay M.A. (2011). State-of-the-art gene-based therapies: the road ahead. Nature Reviews Genetics, 12(5), 316–328.
[7]: Doudna J.A., Charpentier E. (2014). The new frontier of genome engineering with CRISPR-Cas9. Science, 346(6213), 1258096.
[8]: Gillmore J.D. et al. (2019). CRISPR-based therapies for rare genetic diseases: progress and challenges. Nature Reviews Genetics, 20(11), 657–675.
[9]: Dunbar C.E. et al. (2018). Gene therapy comes of age. Science, 359(6372), eaan4672.
[10]: High K.A., Roncarolo M.G. (2019). Gene therapy. The New England Journal of Medicine, 381, 455–464.
[11]: Ormond K.E. et al. (2017). Human germline genome editing. The American Journal of Human Genetics, 101(2), 167–176.
[12]: Cyranoski D., Ledford H. (2018). Genome-edited babies: scientists’ concerns. Nature, 563(7733), 607–608.
[13]: Carroll D. (2020). Genome editing: past, present, and future. Yale Journal of Biology and Medicine, 93(3), 321–328.

This work is licensed under a Creative Commons Attribution 4.0 International License.