Retroviral vectors.
Retroviral vectors are widely used for preclinical and clinical applications. Unlike many of the other types of vectors currently being developed for gene therapy, retroviral vectors are able to genetically modify cells stably without perturbing cell growth. Retroviral vectors based on murine retroviruses are well suited for ex vivo applications where the cells are rapidly dividing. In particular, retroviral viral vectors have been used for a variety of ex vivo gene therapy approaches for treating genetic diseases such as Gaucher and severe combined immunodeficiency (SCID) and for acquired diseases such as cancer and arthritis. However, recent advances in the production of retroviral vectors have allowed for their use in vivo such as for the treatment of cancer and human immunodeficiency virus. The ability to target retroviral vectors to specific cell types will also increase the utility of high titer retroviral vectors for in vivo applications. Clearly retroviral vectors have been extremely useful for both preclinical and clinical gene therapy studies, and it is likely that they will continue to be utilized for ex vivo and in vivo strategies in the future.
Kim
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SH
Department of Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, Pennsylvania, USA.
Kim
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Robbins
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PD
D000818:Animals; D002460:Cell Line; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors; D006801:Humans; D012190:Retroviridae / Q000235:genetics* / Q000502:physiology*