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Validation of a Generation 4 Phosphorus-Containing Polycationic Dendrimer for Gene Delivery Against HIV-1 Briz, Veronica Serramia, María Jesús Madrid González, Ricardo Hameau, Aurélien Caminade, Anne-Marie Majoral, Jean Pierre Muñoz-Fernández, María Ángeles Gene therapy, in which oligomeric genetic material is carried into cells by nano-sized gene delivery vehicles to interfere with gene expression, represents a promising approach for preventive therapy against HIV/AIDS pandemic. Herein, we evaluate the usefulness of a phosphorus-containing dendrimer G4(NH+Et2Cl-)96 as a delivery agent of ODNs and siRNAs. G4(NH+Et2Cl-)96 formed stable complexes with ODNs or siRNAs and exhibited very low cytotoxicity in Sup T1 cells or PBMC. Functional validation was performed by using specific siRNA against HIV-1 Nef, siNEF to interfere in HIV-1 replication. G4(NH+Et2Cl-)96/siNEF dendriplex showed a high efficiency in Nef silencing. Furthermore, in vitro treatment of HIV-infected PBMC with G4(NH+Et2Cl-)96/siNEF dendriplex significantly reduced the viral replication. Our results prove the usefulness of phosphorus-containing dendrimers to deliver and transfect siRNA into CD4-T cells as a potential alternative therapy in the HIV-1 infection. 2024-02-02T14:39:35Z 2024-02-02T14:39:35Z 2012 journal article Briz, V., et al. «Validation of a Generation 4 Phosphorus-Containing Polycationic Dendrimer for Gene Delivery Against HIV-1». Current Medicinal Chemistry, vol. 19, n.o 29, pp. 5044-51. https://doi.org/10.2174/0929867311209025044. 0929-8673 10.2174/0929867311209025044 https://hdl.handle.net/20.500.14352/98366 1875-533X https://doi.org/10.2174/0929867311209025044 eng restricted access Bentham Science Publishers