9 - Dendrimers in gene delivery

Abstract

Gene delivery is an inclusive step of gene therapy. Gene therapy can be defined as the technique for treating the genetic disorders by the transmitting the genetic material into the defective cells of the patient. Gene transfection should be specifically targeted and highly efficient; and it is achieved either by viral or non-viral methods. Although the viral vectors are efficient, the strategy is nonspecific, carcinogenic and immunogenic in vivo. On the contrary, non-viral gene carriers such as dendrimers exhibit higher safety and control providing great potential for gene therapy. Dendrimer complexes with nucleic acid to form dendriplex. Spherical architecture and polyvalency make dendrimer a suitable platform for gene delivery. Dendrimer helps in complexing gene along with providing stability and higher transfection efficiency in vitro and in vivo. Dendrimer condenses nucleic acids into small nanoparticles by ionic interactions and prevents the therapeutic gene from endosomal and nuclear degradation. This chapter illustrates the various aspects of delivery of gene using dendrimer as a carrier.