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<article article-type="research-article" dtd-version="1.3" xmlns:mml="http://www.w3.org/1998/Math/MathML" xmlns:xlink="http://www.w3.org/1999/xlink" xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" xml:lang="ru"><front><journal-meta><journal-id journal-id-type="publisher-id">regmedjournal</journal-id><journal-title-group><journal-title xml:lang="ru">Регенерация органов и тканей</journal-title><trans-title-group xml:lang="en"><trans-title>Регенерация органов и тканей</trans-title></trans-title-group></journal-title-group><issn pub-type="epub">2949-5938</issn><publisher><publisher-name>Общество регенеративной медицины</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="doi">10.60043/2949-5938-2023-1-16-24</article-id><article-id custom-type="elpub" pub-id-type="custom">regmedjournal-4</article-id><article-categories><subj-group subj-group-type="heading"><subject>Research Article</subject></subj-group><subj-group subj-group-type="section-heading" xml:lang="ru"><subject>ОБЗОРЫ И КОММЕНТАРИИ</subject></subj-group><subj-group subj-group-type="section-heading" xml:lang="en"><subject>REVIEWS AND COMMENTS</subject></subj-group></article-categories><title-group><article-title>Три десятилетия развития генной терапии: вехи и перспективы</article-title><trans-title-group xml:lang="en"><trans-title>Three decades of gene therapy development: milestones and prospects</trans-title></trans-title-group></title-group><contrib-group><contrib contrib-type="author" corresp="yes"><name-alternatives><name name-style="eastern" xml:lang="ru"><surname>Макаревич</surname><given-names>П. И.</given-names></name><name name-style="western" xml:lang="en"><surname>Makarevich</surname><given-names>P. I.</given-names></name></name-alternatives><bio xml:lang="ru"><p>Макаревич Павел Игоревич — к.м.н., зав. лабораторией генно-клеточной терапии, Институт регенеративной медицины Медицинского научно-образовательного центра; доцент кафедры биохимии и молекулярной медицины Факультета фундаментальной медицины</p><p>119192, г. Москва, Ленинские горы, 1</p></bio><bio xml:lang="en"><p>Pavel. I. Makarevich — MD, PhD, Head of Gene and Cell Therapy Laboratory, Institute for Regenerative Medicine, Medical Research and Education Center; assistant professor at Department of Biochemistry and Molecular Medicine, Faculty of Medicine</p><p>119192, Moscow, Leninskiye Gory, 1</p></bio><email xlink:type="simple">makarevichpi@my.msu.ru</email><xref ref-type="aff" rid="aff-1"/></contrib></contrib-group><aff-alternatives id="aff-1"><aff xml:lang="ru"><institution>ФГБОУ ВО «Московский государственный университет имени М.В. Ломоносова»</institution><country>Россия</country></aff><aff xml:lang="en"><institution>Lomonosov Moscow State University</institution><country>Russian Federation</country></aff></aff-alternatives><pub-date pub-type="collection"><year>2023</year></pub-date><pub-date pub-type="epub"><day>31</day><month>05</month><year>2023</year></pub-date><volume>1</volume><issue>1</issue><fpage>16</fpage><lpage>24</lpage><permissions><copyright-statement>Copyright &amp;#x00A9; Макаревич П.И., 2023</copyright-statement><copyright-year>2023</copyright-year><copyright-holder xml:lang="ru">Макаревич П.И.</copyright-holder><copyright-holder xml:lang="en">Makarevich P.I.</copyright-holder><license xml:lang="ru" license-type="creative-commons-attribution" xlink:href="https://creativecommons.org/licenses/by/4.0/" xlink:type="simple"><license-p>Данная работа распространяется под лицензией Creative Commons Attribution 4.0.</license-p></license><license xml:lang="en" license-type="creative-commons-attribution" xlink:href="https://creativecommons.org/licenses/by/4.0/" xlink:type="simple"><license-p>This work is licensed under a Creative Commons Attribution 4.0 License.</license-p></license></permissions><self-uri xlink:href="https://www.regmed-journal.ru/jour/article/view/4">https://www.regmed-journal.ru/jour/article/view/4</self-uri><abstract><p>За тридцать лет развития с момента первого применения в клинике генная терапия (ГТ) прошла путь от экспериментального направления до наиболее активно развивающейся области современной биомедицины. Значительные успехи чередовались в течение этого периода с резкими спадами, и именно они вкупе с новыми идеями сформировали основные вехи, которым посвящено данное сообщение. Анализ накопленного опыта формирует направления, лежит в основе открывающихся перспектив развития ГТ и задает вектор для их реализации. При этом нельзя не забывать и о важных вопросах этичности и безопасности, которые являются краеугольными камнями, поддерживающими возможность применения методов ГТ у человека. Настоящее сообщение кратко суммирует наиболее важные события и фундаментальные положения, которые сформировали современный облик ГТ, излагает ее перспективы и проблемные вопросы, которые стоят перед областью в настоящее время.</p></abstract><trans-abstract xml:lang="en"><p>Within past 30 years of progress since its first clinical use, gene therapy (GT) has gone from an experimental field to the most actively developing area of modern biomedicine. Significant advances during this period were mixed with sharp declines, and this pathway taken together with new ideas and concepts has formed the main milestones which this short review addresses. Analysis of accumulated experience provides directions, makes up for emerging prospects for the development of GT and sets the vector for their implementation. At the same time, we should not forget about the crucial issues of ethics and safety, which are the cornerstones supporting the use of GT in human. This report briefly summarizes the most important events and fundamental provisions that have shaped the modern landscape of GT, outlines its prospects and problematic issues in the field.</p></trans-abstract><kwd-group xml:lang="ru"><kwd>генная терапия</kwd><kwd>вектор</kwd><kwd>наследственные заболевания</kwd><kwd>плазмида</kwd><kwd>вирусный вектор</kwd></kwd-group><kwd-group xml:lang="en"><kwd>gene therapy</kwd><kwd>vector</kwd><kwd>hereditary diseases</kwd><kwd>plasmid</kwd><kwd>viral vector</kwd></kwd-group><funding-group><funding-statement xml:lang="ru">Работа выполнена в рамках государственного задания МГУ имени М.В. Ломоносова и по перспективному направлению научно-образовательного развития Московского университета «Молекулярные технологии живых систем и синтетическая биология».</funding-statement><funding-statement xml:lang="en">The work was carried under state assignment of M.V. Lomonosov Moscow State University and the prospective direction of scientific and educational development of M.V. Lomonosov Moscow State University “Molecular technologies of living systems and synthetic biology”.</funding-statement></funding-group></article-meta></front><back><ref-list><title>References</title><ref id="cit1"><label>1</label><citation-alternatives><mixed-citation xml:lang="ru">Anguela X.M., High K.A. Entering the Modern Era of Gene Therapy. Annu Rev Med. 2019;70:273–288.</mixed-citation><mixed-citation xml:lang="en">Anguela X.M., High K.A. Entering the Modern Era of Gene Therapy. Annu Rev Med. 2019;70:273–288.</mixed-citation></citation-alternatives></ref><ref id="cit2"><label>2</label><citation-alternatives><mixed-citation xml:lang="ru">Bulaklak K., Gersbach C.A. The once and future gene therapy. Nat Commun. 2020;11(1):5820.</mixed-citation><mixed-citation xml:lang="en">Bulaklak K., Gersbach C.A. The once and future gene therapy. 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