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Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles <i>in vivo</i>

2023-07-26

Abstract excerpt

Recently approved adeno-associated viral (AAV) vectors for liver monogenic diseases hemophilia A and B are exemplifying the success of liver-directed viral gene therapy. In parallel, additional strategies are rapidly emerging to overcome some inherent AAV limitations, such as non-persistence of episomal transgene in rapidly growing liver and immune response. Integrating lentiviral vectors and non-viral lipid nanop...

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Identifiers and source

Literature Corpus work
18695f76-a85b-5f62-ac2f-c517ca9cd879
DOI
10.1101/2023.07.26.550697
Open publication

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Macrophage inhibitor clodronate enhances liver transduction of lentiviral but not AAV vectors or mRNA lipid nanoparticles <i>in vivo</i>DOI 10.1101/2023.07.26.550697
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