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GP64-pseudotyped lentiviral vectors target liver endothelial cells and correct hemophilia A mice

29 Apr 2024

Abstract excerpt

Lentiviral vectors (LV) are efficient vehicles for in vivo gene delivery to the liver. LV integration into the chromatin of target cells ensures their transmission upon proliferation, thus allowing potentially life-long gene therapy following a single administration, even to young individuals. The glycoprotein of the vesicular stomatitis virus (VSV.G) is widely used to pseudotype LV, as it confers broad tropism...

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PMID: 38684862
DOI: 10.1038/s44321-024-00072-8

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