ABSTRACT
Successful gene therapy is dependent upon the development of safe and efficient gene vectors. Significant advances in nanotechnology will soon perfect the preparation of such DNA nanoparticles. Chapter 5 presents a brief history of gene therapy. This section covers from the beginning, a description of “transforming principle” in the 1930s, to the approbation of the first protocol used in human beings in the 1990s. In the following section, mechanisms for crossing cell barriers and some considerations about the relation of nucleic acids introduced into the cell and its intracellular machinery are described. Then, a simple classification of the different gene therapy approaches and a description of the vector types used are shown. Among non-viral vectors, liposomes, liquid crystals, and associations of lipid nanoparticles or polymers nanoparticles with DNA are described. In this context, nanotechnology has taken an overriding place in the development of non-viral formulations to deliver “genetic drugs”. In this regard, it is revealed that substantial advances in this field, soon, will expand the groundwork of nanostructures based on nucleic acids that have been projected as alternatives to viruses to incorporate specific genes into target cells.
