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• W2023184276 abstract "The use of retroviral vectors has shown an actual clinical benefit in a few inherited diseases. However, the occurrence of cases of leukemia after the X-SCID gene therapy trial raised concerns about the safety of insertional mutagenesis inherent to the biology of the retrovirus. Although the retrovirus has long been known to integrate into the host chromosome, and thus have the potential to activate the nearby gene, there has been no convenient method of studying or assaying such a cis-activation phenomenon.In the present study, we report an in vitro assay system in which the effect of retroviral integration on the expression of the neighbouring gene can be studied. In this system, a retroviral vector and the neighbouring reporter gene were constructed in a single plasmid as if it had integrated into the chromosome.Using this assay, we found that the full-length long terminal repeat (LTR) could indeed activate the neighbouring gene expression from a distance and the magnitude of its activation was highly increased when this LTR was placed in the vicinity of the transcription start site of the gene, whereas the truncated LTR exerted little influence.This assay system might provide a useful tool for selecting the appropriate vector structure, as well as for studying the molecular mechanism underlying the cis-activation by the viral LTR." @default.
• W2023184276 created "2016-06-24" @default.
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• W2023184276 date "2008-08-01" @default.
• W2023184276 modified "2023-09-22" @default.
• W2023184276 title "Development of anin vitrocell culture assay system for measuring the activation of a neighbouring gene by the retroviral vector" @default.
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• W2023184276 doi "https://doi.org/10.1002/jgm.1213" @default.
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• W2023184276 hasPublicationYear "2008" @default.
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