Recent Advances in Nanocarriers Used for Selective Gene Silencing Therapy

Gene therapy has been proven to be a promising tool to treat chronic and acquired diseases because it allows us to regulate a gene sequence by repairing, replacing, or silencing a target gene. In this chapter, gene therapy involving gene silencing by RNA interference (RNAi) will be presented and discussed, as well as its advantages and disadvantages. Furthermore, it will also present nanocarriers for the topical and systemic administration of small interfering RNA (siRNA, the RNA effector molecule) and the advantages and disadvantages of each system will be addressed. Recent clinical and preclinical advances involving delivery systems for siRNA application will be emphasized. The chapter will end by addressing the future perspectives of the specific knockdown of therapeutic targets by RNA interference.