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- W2261865673 abstract "Retroviral vector gene therapy is a promising approach to treating HIV-1. However, integrated vectors are mutagens with the potential to dysregulate nearby genes and cause severe adverse side effects. Leukemia has already been a documented severe adverse event in gene therapy clinical trials for the treatment of primary immunodeficiencies. These side effects will need to be reduced or avoided if retroviral vectors are to be used clinically for HIV-1 treatment. The addition of chromatin insulators to retroviral vectors is a potential strategy for reducing adverse side effects. Insulators have already been effectively used in retroviral vectors to reduce genotoxicity in pre-clinical studies. Here, we will review how insulators function, genotoxicity in gene therapy clinical trials, the design of insulated retroviral vectors, promising results from insulated retroviral vector studies, and considerations for the development of insulated retroviral treatment vectors for HIV-1 gene therapy." @default.
- W2261865673 created "2016-06-24" @default.
- W2261865673 creator A5065033882 @default.
- W2261865673 creator A5079548188 @default.
- W2261865673 date "2016-02-02" @default.
- W2261865673 modified "2023-10-17" @default.
- W2261865673 title "Insulators to Improve the Safety of Retroviral Vectors for HIV Gene Therapy" @default.
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- W2261865673 doi "https://doi.org/10.3390/biomedicines4010004" @default.
- W2261865673 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5344246" @default.
- W2261865673 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/28424756" @default.
- W2261865673 hasPublicationYear "2016" @default.
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