Novel non-viral gene delivery systems composed of carbosilane dendron functionalized nanoparticles prepared from nano-emulsions as non-viral carriers for antisense oligonucleotides

Int J Pharm. 2015 Jan 15;478(1):113-123. doi: 10.1016/j.ijpharm.2014.11.031. Epub 2014 Nov 15.

Abstract

The development of novel and efficient delivery systems is often the limiting step in fields such as antisense therapies. In this context, poly(d,l-lactide-co-glycolide) acid (PLGA) nanoparticles have been obtained by a versatile and simple technology based on nano-emulsion templating and low-energy emulsification methods, performed in mild conditions, providing good size control. O/W polymeric nano-emulsions were prepared by the phase inversion composition method at 25°C using the aqueous solution/polysorbate80/[4 wt% PLGA in ethyl acetate] system. Nano-emulsions formed at oil-to-surfactant (O/S) ratios between 10/90-90/10 and aqueous contents above 70 wt%. Nano-emulsion with 90 wt% of aqueous solution and O/S ratio of 70/30 was chosen for further studies, since they showed the appropriate characteristics to be used as nanoparticle template: hydrodynamic radii lower than 50 nm and enough kinetic stability. Nanoparticles, prepared from nano-emulsions by solvent evaporation, showed spherical shape, sizes about 40 nm, negative surface charges and high stability. The as-prepared nanoparticles were functionalized with carbosilane cationic dendrons through a carbodiimide-mediated reaction achieving positively charged surfaces. Antisense oligonucleotides were electrostatically attached to nanoparticles surface to perform gene-silencing studies. These complexes were non-haemolytic and non-cytotoxic at the concentrations required. The ability of the complexes to impart cellular uptake was also promising. Therefore, these novel nanoparticulate complexes might be considered as potential non-viral carriers in antisense therapy.

Keywords: Antisense therapy; Cationic dendron; Nano-emulsion; Non-viral gene delivery systems; PIC emulsification method; PLGA nanoparticles.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • Cell Survival / drug effects
  • Dendrimers* / administration & dosage
  • Dendrimers* / chemistry
  • Emulsions
  • Erythrocytes / drug effects
  • Erythrocytes / pathology
  • Gene Transfer Techniques*
  • Hemolysis / drug effects
  • Humans
  • Lactic Acid / chemistry
  • Luciferases, Renilla / genetics
  • Nanoparticles* / administration & dosage
  • Nanoparticles* / chemistry
  • Oligonucleotides, Antisense / administration & dosage*
  • Oligonucleotides, Antisense / chemistry
  • Polyglycolic Acid / chemistry
  • Polylactic Acid-Polyglycolic Acid Copolymer
  • Silanes* / chemistry
  • Static Electricity

Substances

  • Dendrimers
  • Emulsions
  • Oligonucleotides, Antisense
  • Silanes
  • carbosilane
  • Polylactic Acid-Polyglycolic Acid Copolymer
  • Polyglycolic Acid
  • Lactic Acid
  • Luciferases, Renilla