Synthetic vectors for genetic drug delivery

Abstract

Gene therapy is a new approach in the treatment of numerous diseases caused by damage to or lack of certain genes. Its application seems to be very effective, especially in the case of cancer diseases, where genetic drugs can be designed to selectively eliminate damaged cells. Due to the chemical properties of nucleic acids, their introduction into the cell is very difficult. DNA is a highly polar and negatively charged molecule unable to cross the plasma membrane. Moreover, "naked DNA" is exposed to enzymatic degradation by nucleases present within cells and biological fluids. Thus, for efficient and selective DNA delivery into the cells, it is necessary to use a suitable carrier system that can protect it from nucleases and enable it to cross the plasma membrane. Cationic lipid- and synthetic polymer-based carriers are very promising tools for gene delivery into different cell types, but there are still many obstacles to overcome before efficient gene therapy using synthetic vectors will be truly viable. Studies on the nature of interaction between genetic drugs and their lipid carriers, as well as on the mechanism of gene delivery into the cell allowed improvements to be made to currently existing synthetic vectors, and newer and more effective systems to be created. It is crucial that the lipid carriers and genetic drugs have a low degree of toxicity and high degree of specificity for effective gene delivery to occur. That is why the efforts of many laboratories are focused on designing and producing new forms of genetic drugs and derivatives of lipids in order to facilitate the creation of suitable gene delivery systems.