ABSTRACT

Gene therapy has gained attention in the last few years because of its efficacy in the treatment of numerous diseases, particularly those caused by oncological and genetic disturbances. The success of gene therapy is completely dependent on the development of nontoxic and highly efficient gene delivery systems. Thus, in order to protect oligonucleotides from damage, facilitate their entry into the target cells, and promote an efficient endosomal release, new lipid- and polymer-based delivery systems have emerged in the past few years. The most promising strategies involving the complexation of nucleic acids with lipids (lipoplexes) and different kinds of polymers (polyplexes) are reviewed in this work. The advantages and disadvantages of each system and the possibility of using polymers and lipids in a same formulation are also explored. Finally, the future perspectives for gene therapy associated with nanotechnology, an approach that seems to be quite promissory, bringing significant new insights into the treatment of numerous diseases are discussed.