%0 Journal Article
%A Briz, Veronica
%A Serramia, María Jesús 
%A Madrid González, Ricardo
%A Hameau, Aurélien
%A Caminade, Anne-Marie
%A Majoral, Jean Pierre
%A Muñoz-Fernández, María Ángeles 
%T Validation of a Generation 4 Phosphorus-Containing Polycationic Dendrimer for Gene Delivery Against HIV-1
%D 2012
%@ 0929-8673
%U https://hdl.handle.net/20.500.14352/98366
%X 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.
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