Non-viral gene therapy offers the potential to deliver nucleic acids producing therapeutic proteins to treat genetic diseases without the limitations observed with viral vectors. Before the therapeutic potential of non-viral gene delivery can be realized, several barriers to efficient gene delivery must be overcome. One delivery barrier of interest is the enhancement of endosomal escape to prev...

Microbubbles and ultrasound have recently been investigated with a view to improving the transfection efficiency of non-viral gene delivery systems. However, microbubbles are unstable and their targeting ability is insufficient for clinical use. To circumvent these problems, we developed novel polyethyleneglycol (PEG) modified liposomes (Bubble liposomes) containing perfluoropropane, which is a...

The primary challenge for gene therapy is to develop a method that delivers a therapeutic gene (transgene) to selected cells where proper gene expression can be achieved. Gene therapy using viral or synthetic vectors is currently one of the most promising strategies for many diseases. Cationic lipid–DNA complexes have emerged as one of the major non-viral DNA delivery tools. An ideal gene deliv...

Chitosan is a naturally occurring polymer derived from the deacetylation of chitin, which an abundant carbohydrate found mainly in shells various marine and terrestrial (micro)organisms. has been extensively used to construct nanoparticles (NPs), are biocompatible, biodegradable, non-toxic, easy prepare, can function as effective drug delivery systems. Moreover, chitosan NPs have employed gene ...

A remarkable non-linearity was found between dose and transfection activities of non-viral gene delivery systems, such as a Lipofectamine/DNA complex and an octaarginine-modified multifunctional envelope-type nano device (R8-MEND). We measured the nuclear delivery of pDNA to distinguish the non-linearity in intracellular pharmacokinetics or pharmacodynamics after transfection with R8-MEND at di...

A major goal of nucleic acid based therapies is to treat inherted and acquired disorders by adding, correcting, suppressing or eplacing genes (Abbas et al., 2008). Advantages of non-viral vectors or delivering nucleic acid based therapies include ease of scale-up, torage stability and improved quality control. The most promisng non-viral vectors have been liposomes and cationic polymers hich co...

A non-viral gene delivery nanovehicle based on Alkyl-PEI2k capped MnO nanoclusters was synthesized via a simple, facile method and used for efficient siRNA delivery and magnetic resonance imaging.

DNA based transposon vectors offer a mechanism for non-viral gene delivery into mamma‐ lian and human cells. These vectors work via a cut-and-paste mechanim whereby transpo‐ son DNA containing a transgene(s) of interest is integrated into chromosomal DNA by a transposase enzyme. The first DNA based transposon system which worked efficienty in human cells was sleeping beauty. This was followed a...

Ocular gene therapy holds promise for the treatment of numerous blinding disorders. Despite the significant progress in the field of viral and non-viral gene delivery to the eye, significant obstacles remain in the way of achieving high-level transgene expression without adverse effects. The retinal pigment epithelium (RPE) is involved in the pathogenesis of retinal diseases and is a key target...