Nonviral Gene Therapy: Design and Application of Inorganic Nanoplexes

Nucleic acid therapies show potential clinical application for different diseases from cystic fibrosis, hemoglobinopathies, and hemophilia, to cancer treatment, cardiovascular disease, and genetic vaccines. The main challenge in obtaining a successful therapy is to find a safe and effective delivery system able to form a stable complex with the carried nucleic acid, protect it from extracellular degradation, target the cell of interest, and release its cargo inside. To this end, nonviral vectors, such as inorganic nanoparticles, are promising candidates. These nanoparticles show several interesting properties, such as tunable size and surface properties, multifunctional ability, and the avoidance of immunogenicity and cytotoxicity problems caused by viral vectors. In this chapter we will focus on the design of inorganic nanoplexes based on gold nanoparticles, quantum dots, superparamagnetic iron oxide nanoparticles, graphene and carbon nanotubes for nucleic acid delivery, and their therapeutic applications and future prospects in the field.