Читать книгу Nanopharmaceutical Advanced Delivery Systems - Группа авторов - Страница 55

Gene therapy is the microlevel therapeutics that works at molecular level by “switching genes on or off” through the use of nucleic acid-based drugs (NABDs), example of which include oligodeoxynucleotides, plasmid DNA, ribozymes, siRNA, miRNA, and related chemically synthesized molecules. As these molecules are biodegradable, so stability of vectors carrying them is indispensible for them to reach the target cell/tissue, which is defective or mutated. Nanoparticulate carriers can play a crucial role in gene therapy by providing safe, non-toxic, non-viral carriers. Recently, United States Food and Drug Administration (FDA) has approved the first ever siRNA-lipid based formulation for human use under the trade name Onpattro^™ for hereditary amyloidosis [97]. Cationic lipids and cationic polymers have also been used for gene therapy by formation of lipoplexes and polyplexes, respectively [98-99]. Lipoplexes are cationic lipid-based non-viral gene delivery systems formed via electrostatic interaction with the negatively charged phosphate groups present in nucleic acids. Similarly, polyplexes are formed using cationic polymers. The methods generally used for the preparation of nanoparticulate carriers can also be used for the preparation of these carriers.