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Macrophage Inhibitor Clodronate Enhances Liver Transduction of Lentiviral but Not Adeno-Associated Viral Vectors or mRNA Lipid Nanoparticles in Neonatal and Juvenile Mice

Recently approved adeno-associated viral (AAV) vectors for liver monogenic diseases haemophilia A and B are exemplifying the success of liver-directed viral gene therapy. In parallel, additional gene therapy strategies are rapidly emerging to overcome some inherent AAV limitations, such as the non-p...

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Principais autores: Loukia Touramanidou, Sonam Gurung, Claudiu A. Cozmescu, Dany Perocheau, Dale Moulding, Patrick F. Finn, Andrea Frassetto, Simon N. Waddington, Paul Gissen, Julien Baruteau
Format: Artigo
Sprog:Inglês
Udgivet: MDPI AG 2024-11-01
Serier:Cells
Fag:
gene therapy
AAV
LNP mRNA
lentiviral vector
LV
macrophages
Online adgang:https://www.mdpi.com/2073-4409/13/23/1979
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