mvjrМедицинский вестник Юга РоссииMedical Herald of the South of Russia2219-80752618-7876The Rostov State Medical Universitymvjr-1252Research ArticleОБЗОРЫREVIEWОсновы безопасности генной терапииBasis of Gene TherapyИпатовС. Е.IpatovS. E.

117997, г. Москва, Ленинский проспект, д. 117/2

РумянцевС. А.RoumiantsevS. A.

117997, г. Москва, Ленинский проспект, д. 117/2

s_roumiantsev@mail.ruФГУ Федеральный научно-клинический центр детской гематологии, онкологии и иммунологии Минздравсоцразвития РоссииРоссияFederal Research Center of Pediatric Hematology, Oncology and ImmunologyRussian Federation2010300620100249Copyright © Ипатов С.Е., Румянцев С.А., 20102010Ипатов С.Е., Румянцев С.А.Ipatov S.E., Roumiantsev S.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.medicalherald.ru/jour/article/view/1252

К настоящему времени в научных кругах всего мира обсуждаются проблемы безопасности при проведении генной терапии у широкой категории пациентов. В данном обзоре суммированы результаты клинических исследований, приведены пояснения относительно побочных эффектов, связанных с генотоксичностью векторной интеграции, обсуждаются факторы, которые могут вызывать случаи генотоксичности, а также предложены подходы, способные сохранить или повысить клиническую эффективность генной терапии с использованием в качестве мишеней гемопоэтических стволовых клеток, при этом существенно снижая риск развития лейкемии и других побочных эффектов, связанных с включением вектора в геном.

By present time safety problems of gene therapy are widely discussed among scientists of all world. In this review results of clinical researches are summarized; explanations concerning side effects of vectors integration are given; factors that can cause genotoxicity are discussed. Approaches which can save or increase clinical efficacy of gene therapy with use as targets hematopoietic stem cells, thus significantly reduced risk of leukemia development and other side effects related to vectors including in genome, are presented.

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