Dendrimers for gene therapy

Gene therapy has emerged as a realistic therapeutic strategy for the treatment of both inherited diseases and acquired diseases such as cancer. Nonviral vectors have attracted much attention as an alternative to viral vectors. Dendrimers are highly branched macromolecules that have been recently employed as nonviral gene delivery vectors. Their unique molecular architecture and key properties such as globular shape and a high density of surface functional groups make these nanoscale materials attractive vehicles for gene delivery. The present chapter covers the structure, synthesis, physicochemical and biological properties of dendrimers as well as some of their applications for gene delivery. The mechanism of gene transfer using dendrimers and some functionalization strategies for overcoming the extracellular and intracellular barriers for efficient delivery of genetic material are discussed. Finally, the use of dendrimers for the delivery of gene therapy in vivo is summarized.