title: [Safety of retroviral vectors in gene therapy].
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.
authors.forename: E V
authors.initials: EV
authors.lastname: Bogoslovskaia
authors.forename: D V
authors.initials: DV
authors.lastname: Glazkova
authors.forename: G A
authors.initials: GA
authors.lastname: Shipulin
authors.forename: V V
authors.initials: VV
authors.lastname: Pokrovskiĭ
mesh_terms: D000818:Animals; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000494:pharmacology*; D006801:Humans; D012190:Retroviridae / Q000235:genetics*; D016511:Severe Combined Immunodeficiency / Q000628:therapy*