avkАрхивъ внутренней медициныThe Russian Archives of Internal Medicine2226-67042411-6564“SINAPS” LLC10.20514/2226-6704-2022-12-5-363-369avk-1508Research ArticleОБЗОРНЫЕ СТАТЬИREVIEW ARTICLESГЕННАЯ ТЕРАПИЯ БОЛЕЗНЕЙ ЧЕЛОВЕКА: ПОСЛЕДНИЕ ДОСТИЖЕНИЯ И БЛИЖАЙШИЕ ПЕРСПЕКТИВЫ РАЗВИТИЯGene Therapy for Human Diseases: Recent Achievements and Near-Term Development Prospectshttps://orcid.org/0000-0003-4973-039XАйтбаевК. А.AitbaevK. A.

Бишкек

Bishkek

https://orcid.org/0000-0001-8513-9279МуркамиловИ. Т.MurkamilovI. T.

Илхом Торобекович Муркамилов

Бишкек

Ilkhom T. Murkamilov

Bishkek

murkamilov.i@mail.ruhttps://orcid.org/0000-0002-7653-0433МуркамиловаЖ. А.MurkamilovaZ. A.

Бишкек

Bishkek

https://orcid.org/0000-0003-0632-6653ЮсуповФ. А.YusupovF. A.

Ош

Osh

Научно-исследовательский институт молекулярной биологии и медициныКыргызстанScientific and research Institute of molecular biology and medicineKyrgyzstanКыргызская государственная медицинская академия имени И.К. Ахунбаева; ГОУ ВПО Кыргызско-Российский славянский университетКыргызстанI.K. Akhunbaev Kyrgyz State Medical Academy; SEI HPE Kyrgyz Russian Slavic UniversityKyrgyzstanГОУ ВПО Кыргызско-Российский славянский университетКыргызстанSEI HPE Kyrgyz Russian Slavic UniversityKyrgyzstanОшский государственный университетКыргызстанOsh State UniversityKyrgyzstan202201102022125363369Copyright © Айтбаев К.А., Муркамилов И.Т., Муркамилова Ж.А., Юсупов Ф.А., 20222022Айтбаев К.А., Муркамилов И.Т., Муркамилова Ж.А., Юсупов Ф.А.Aitbaev K.A., Murkamilov I.T., Murkamilova Z.A., Yusupov F.A.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://www.medarhive.ru/jour/article/view/1508

В статье кратко изложены недавние успехи в генетической медицине, которые проложили путь для дальнейшего развития генной терапии и заложили основу для разработки технологий следующего поколения. Рассмотрены вопросы, связанные с основным препятствием для более широкого применения методов генной терапии, в частности, с иммунным ответом на векторы доставки генов и продукты чужеродных трансгенов. В этом контексте обсуждается роль новых технологий, позволяющих обойти иммунное препятствие, таких как разработка модифицированных капсидов адено-ассоциированых вирусов (AAV) и методов временного удаления антител из кровотока, а также переноса гена в ткани с помощью наночастиц. Наряду с технологиями первого поколения генной терапии, ориентированных на доставку трансгенов в ткани-мишени, резюмируются последние достижения в разработке совершенно нового подхода к генной терапии, основанного на точной модификации последовательностей генома человека — технологии редактирования генов. И наконец, обозначены перспективные технологии редактирования генов следующего поколения, такие как технологии редактирования, нацеленные на РНК и технологии редактирования эпигенома, которые являются более специфичными и точными, эффективными и применимыми к различным группам заболеваний. В заключение делается вывод, что генная терапия является на сегодняшний день самой захватывающей и революционной биотехнологией современности как из-за недавнего прогресса, так и из-за возможностей, которые она может обеспечить в ближайшем будущем.

The article briefly summarizes recent advances in genetic medicine that paved the way for the further development of gene therapy and set the stage for the development of next generation technology. Issues related to the main obstacle for wider application of gene therapy methods, in particular, with the immune response to gene delivery vectors and transgene products are considered. In this context, the role of new technology allowing to bypass the immune obstacle, such as development of modified capsids of adeno-associated viruses (AAV) and methods for temporary removal of antibodies from the bloodstream, as well as gene transfer into tissues using nanoparticles, is discussed. Along with the technology of the first generation gene therapy focused on the delivery of transgenes into target tissues, latest advances in the development of a completely new approach to gene therapy which is based on precise modification of the human genome sequence, gene editing technology, are summarized. Finally, promising next-generation gene editing technology is outlined, such as RNA-targeted editing technology and epigenome editing technology, which are more specific, precise, efficient and applicable to different groups of diseases. The article concludes that gene therapy and, in particular, human genome editing is perhaps the most exciting and revolutionary biotechnology of our time, due to both recent developments and opportunities it might provide in the nearest future.

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The authors declare that there are no conflicts of interest present.