Carbosilane dendrimers as gene delivery agents for the treatment of HIV infection

J Control Release. 2014 Jun 28:184:51-7. doi: 10.1016/j.jconrel.2014.03.048. Epub 2014 Apr 8.

Abstract

Despite the use of siRNA in the downregulation of HIV-1 replication which has been reported, CD4 T lymphocytes are difficult to transfect with non-viral vectors. We determined whether second generation carbosilane dendrimers (2G-NN16 and 2G-03NN24) may be efficient transfectants in CD4 T lymphocytes. Dendrimers were also tested on macrophages to determine whether they can modify macrophage phenotype and induce an inflammatory response. The nanoconjugate formed by 2G-03NN24/siRNA-Nef presents the highest inhibition of HIV-1 replication. Dendrimers presented safety properties because they did not induce proliferation on CD4 T lymphocytes and decrease the release of TNFα and IL-12p40 by macrophages. Both dendrimers also decrease the phagocytosis activity. Additionally, 2G-03NN24 dendrimer decreases the CCL2 and CCR2 expression in macrophages. Carbosilane dendrimers 2G-NN16 and 2G-03NN24 can be used as efficient non-viral vectors for gene therapy applications, mainly in the treatment of HIV infection.

Keywords: CD4 lymphocytes and macrophages; Carbosilane dendrimers; Gene therapy; HIV infection; siRNA delivery.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • CD4-Positive T-Lymphocytes / drug effects
  • CD4-Positive T-Lymphocytes / immunology
  • CD4-Positive T-Lymphocytes / virology
  • Cell Survival / drug effects
  • Cells, Cultured
  • Cytokines / immunology
  • Dendrimers / administration & dosage*
  • Gene Products, nef / genetics
  • Gene Transfer Techniques*
  • HIV Infections / drug therapy*
  • HIV-1 / drug effects
  • HIV-1 / physiology
  • Humans
  • Leukocytes, Mononuclear
  • Lipopolysaccharides
  • Macrophages / drug effects
  • Macrophages / immunology
  • Organosilicon Compounds / administration & dosage*
  • Phagocytosis / drug effects
  • RNA, Small Interfering / administration & dosage*
  • Silanes / administration & dosage*
  • Virus Replication / drug effects

Substances

  • 2G-03NN24
  • 2G-NN16
  • Cytokines
  • Dendrimers
  • Gene Products, nef
  • Lipopolysaccharides
  • Organosilicon Compounds
  • RNA, Small Interfering
  • Silanes