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In this review we introduce a new concept for developing a nonviral gene delivery system which we call ˵Programmed Packaging.″ Based on this concept, we succeeded in developing a multifunctional envelope-type nano device (MEND), which exerts high transfection activities equivalent to those of an adenovirus in a dividing cell. The use of MEND has been extended to in vivo applications. PEG/peptide/DOPE ternary conjugate (PPD)-MEND, a new in vivo gene delivery system for the targeting of tumor cells that dissociates surface-modified PEG in tumor tissue by matrix metalloproteinase (MMP) and exerts significant transfection activities, was developed. In parallel with the development of MEND, a quantitative gene delivery system, Confocal Image-assisted 3-dimensionally integrated quantification (CIDIQ), also was developed. This method identified the rate-limiting step of the nonviral gene delivery system by comparing it with adenoviral-mediated gene delivery. The results of this analysis provide a new direction for the development of rational nonviral gene delivery systems. [ABSTRACT FROM AUTHOR]

Motivated by the promises of gene therapy, there is great interest in developing non-viral lipid-based vectors for therapeutic applications due to their low immunogenicity, low toxicity, ease of production, and the potential of transferring large pieces of DNA into cells. In fact, cationic liposome (CL) based vectors are among the prevalent synthetic carriers of nucleic acids (NAs) currently used in gene therapy clinical trials worldwide. These vectors are studied both for gene delivery with CL–DNA complexes and gene silencing with CL–siRNA (short interfering RNA) complexes. However, their transfection efficiencies and silencing efficiencies remain low compared to those of engineered viral vectors. This reflects the currently poor understanding of transfection-related mechanisms at the molecular and self-assembled levels, including a lack of knowledge about interactions between membranes and double stranded NAs and between CL–NA complexes and cellular components. In this review we describe our recent efforts to improve the mechanistic understanding of transfection by CL–NA complexes, which will help to design optimal lipid-based carriers of DNA and siRNA for therapeutic gene delivery and gene silencing. [ABSTRACT FROM AUTHOR]

The successful application of gene therapy through DNA transfection into the cell is still a great challenge in ongoing research. Hyperbranched polyamines are highly branched macromolecules, and have gained significant attention in the last two decades, due to their relative ease of preparation, their shape, and their multi-functionality. This review deals with the syntheses of various hyperbranched polyamines that are prepared through a one-step polymerization process. Furthermore, we present the current status of polyamines as gene carriers and describe their versatility, and their properties such as structure-property dependency, gene transfection efficiency, and cytotoxicity profiles of hyperbranched polyamines. [ABSTRACT FROM AUTHOR]

In this study; poly(n-isopropylacrylamide) P(NIPA) based nanoparticles were prepared by dispersion polymerization technique. Prepared nanoparticles were characterized by their morphology and chemical point of view using different techniques. Morphological evaluations of the nanoparticles were taken by using an atomic force microscope (AFM). Zeta potential and the particle size of NIPA based nanoparticles in aqueous solutions were determined with DLS (Dynamic Light Scattering) technique at different pHs and different temperatures. MTT studies were carried out to confirm the non-toxic character of the nanoparticles. In the transfection and expression studies; the plasmid DNA for Green Fluorescence Protein (GFP) expressing was used as a model plasmid DNA and the HeLa cells were used as the model cell line. [ABSTRACT FROM AUTHOR]