SAFETY OF RETROVIRAL VECTORS IN GENE THERAPY

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Abstract

Retroviral vectors are widely used in gene therapy and found to be an effective tool for the delivery of genetic constructs into cells. A unique feature of these vectors is the ability to incorporate therapeutic genes into a chromosome that ensures its passage to all progeny cells and enables to cure the diseases requiring genetic correction of dividing cells such as hematopoietic cells or skin cells. Retroviral vectors have been successfully used in gene therapy clinical trials for the treatment of 2 forms of severe combined immunodeficiencies and some other hereditary blood disorders. However, the integration of the vector into the chromosome was accompanied by genotoxicity and caused development of hematologic malignancies in several patients. Later it was shown that genotoxicity is not a general feature of retroviral vectors but it depends on many factors. In the present article we discuss safety issues concerning the use of different retroviral vectors in gene therapy. The description of modern vectors which designed to avoid the genotoxicity and other possible side effects are given.

 

About the authors

E. V. Bogoslovskaya

Central Research Institute of Epidemiology, Moscow

Author for correspondence.
Email: elena.bogoslovskaya@pcr.ru
кандидат медицинских наук, старший научный сотрудник отдела молекулярной диагностики и эпидемиологии ФБУН ЦНИИ эпидемиологии Роспотребнадзора Адрес: 111123, Москва, ул. Новогиреевская, д. 3А Тел.: (495) 974-96-46, факс: (495) 305-54-23 Russian Federation

D. V. Glazkova

Central Research Institute of Epidemiology, Moscow

Email: glazkova@pcr.ru
кандидат биологических наук, научный сотрудник отдела молекулярной диагностики и эпидемиологии ФБУН ЦНИИ эпидемиологии Роспотребнадзора Адрес: 111123, Россия, Москва, ул. Новогиреевская, д. 3А Тел.: (495) 974-96-46, (916) 958-09-57, факс: (495) 305-54-23 Russian Federation

G. A. Shipulin

Central Research Institute of Epidemiology, Moscow

Email: german@pcr.ru
кандидат медицинских наук, заведующий отделом молекулярной диагностики и эпидемиологии ФБУН ЦНИИ эпидемиологии Роспотребнадзора Адрес: 111123, Россия, Москва, ул. Новогиреевская, д. 3А Тел.: (495) 974-96-46, факс: (495) 305-54-23 Russian Federation

V. V. Pokrovskii

Central Research Institute of Epidemiology, Moscow

Email: info@pcr.ru
доктор медицинских наук, академик РАМН, заместитель директора ФБУН ЦНИИ эпидемиологии Роспотребнадзора по научной работе Адрес: 111123, Россия, Москва, ул. Новогиреевская, д. 3А Тел.: (495) 974-96-46, факс: (495) 305-54-23 Russian Federation

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