Hydrodynamic gene delivery (HGD) is an established method of the last decade where macromolecules, non-normally permeable to cell membrane, are delivered intracellular. The basic principle is that a large volume of solution, containing genes or oligonucleotides, RNA, proteins or other compounds, is infused rapidly in circulation to permit the entrance of these substances to parenchymal cells. E...

BACKGROUND Hydrodynamic injection is an effective method for DNA delivery in mouse liver and is being translated to larger animals for possible clinical use. Similarly, phiC31 integrase has proven effective in mediating long-term gene therapy in mice when delivered by hydrodynamic injection and is being considered for clinical gene therapy applications. However, chromosomal aberrations have bee...

background: the poor permeability of the plasma and nuclear membranes to dna plasmids are two major barriers for the development of these therapeutic molecules. therefore, success in gene therapy approaches depends on the development of efficient and safe non-viral delivery systems. objectives: the aim of this study was to investigate the in vitro delivery of plasmid dna encoding hpv16 e7 gene ...

RNA interference (RNAi) is an exciting new tool to effect acute in vivo knockdown of genes for pharmacological target validation. Testing the application of this technology to metabolic disease targets, three RNAi delivery methods were compared in two frequently utilized preclinical models of obesity and diabetes, the diet-induced obese (DIO) and B6.V-Lep/J (ob/ob) mouse. Intraperitoneal (i...

A hemodynamic study of hydrodynamic gene delivery (HGD) from the tail vein in rodents has inspired a mechanism and an approach to further improve the efficacy of this procedure. However, there is no report on the hemodynamics of a regional HGD, which is an inevitable approach in large animals. Here, we report the hemodynamics of a regional hydrodynamic injection in detail based on 3D volume dat...

The safe, targeted and effective delivery of gene therapeutics remains a significant barrier to their broad clinical application. Here we develop a magnetic nucleic acid delivery system composed of iron oxide nanoparticles and cationic lipid-like materials termed lipidoids. Coated nanoparticles are capable of delivering DNA and siRNA to cells in culture. The mean hydrodynamic size of these nano...

Chapter 1 presents a review of the current literature on non-viral gene delivery with special emphasis on peptide gene delivery carriers and the use of acridine. Barriers to systemically-administered non-viral gene delivery and remedies to overcome them individually will be discussed, and two physical methods of particular importance in examining circulating polyplexes (DNA and peptide carrier)...