Details

Autor(en) / Beteiligte

• Briz, V
• Serramía, M J
• Madrid, R
• Hameau, A
• Caminade, Anne-Marie
• Majoral, J P
• Muñoz-Fernández, M A

Titel

Validation of a generation 4 phosphorus-containing polycationic dendrimer for gene delivery against HIV-1

Ist Teil von

• Current medicinal chemistry, 2012-10, Vol.19 (29), p.5044-5051

Ort / Verlag

United Arab Emirates

Erscheinungsjahr

2012

Quelle

MEDLINE

Beschreibungen/Notizen

• 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.