OncohematologyOncohematologyОнкогематология1818-83462413-4023Publishing House ABV Press663BASIC RESEARCHФУНДАМЕНТАЛЬНЫЕ ИССЛЕДОВАНИЯ В ПРАКТИЧЕСКОЙ МЕДИЦИНЕ НА СОВРЕМЕННОМ ЭТАПЕSafety issues of gene therapyВопросы безопасности генной терапииIpatovS. E.ИпатовС. Е.
Russian Federation

Moscow

ФГУ Федеральный научно-клинический центр детской гематологии, онкологии и иммунологии

Москва

RoumiantsevS. A.РумянцевС. А.
Russian Federation

Moscow

Сергей Александрович Румянцев

ФГУ Федеральный научно-клинический центр детской гематологии, онкологии и иммунологии

Москва

s.roumiantsev@hiidg.ruFederal Research Center of Pediatric Hematology, Oncology and ImmunologyМинздравсоцразвития России230220101576323112022231120222010https://oncohematology.abvpress.ru/ongm/article/view/663

   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.

   В настоящее время в научных кругах всего мира широко 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, которые позволят сохранить или повысить клиническую эффективность генной терапии с использованием в качестве мишеней гемопоэтических стволовых клеток при существенном снижении риска развития лейкемии и других побочных эффектов, связанных с включением вектора в геном.

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