Gene therapy is a novel approach to treat, cure, or ultimately prevent disease by changing the expression of a persons genes. Several cationic polymers have been investigated for their functionality in non-viral gene delivery systems; however, most of these polymers are synthetic, not biocompatible and impose toxicity problems. Chitosans, which are linear cationic polysaccharides, have been investigated as a biocompatible and non-toxic alternative for delivery of genes and other nucleic acids, both in vitro and in vivo1 (using PROTASAN ultrapure chitosans). It has been shown that low molecular weight chitosans (4-6 kDa) with a narrow molecular weight distribution have very favorable properties in non-viral gene delivery systems.
NOVAFECT O are a family of proprietary oligomeric chitosans that are soluble at neutral pH, they are not viscous and easily form polyplexes with nucleic acids through electrostatic interactions. The formation of polyplexes can be shown by measuring their mobility in an agarose gel assay, and polyplexes can be visualized using Atomic Force Microscopy. Non-aggregated polyplexes are formed of rod-like, toroidal and spherical shapes, typically of the order of 50 nm.
A subtle balance between the stability and dissociation of NOVAFECT-DNA polyplexes is one of the key determinants of the transfection efficiency. Several studies indicate that a high degree of deacetylation, and a narrow molecular weight distribution is important for optimum gene delivery both in vitro and in vivo.
In vitro studies have shown that linear NOVAFECT O can efficiently transfect epithelial human embryonic kidney 293 cell line, epithelial Calu-3 cells, and cervix epithelial HeLa cells, with no acute toxicity observed. Efficient in vivo luciferase gene expression in mouse lungs has also been demonstrated following intratracheal administration, with no acute toxicity observed. Non-viral gene delivery using NOVAFECT O appears safe and non-toxic, and represents an attractive alternative to other non-viral and viral vector based gene delivery systems.
NOVAFECT-siRNA kit to be included
For more information on NOVAFECT click here
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