RNA delivery for cancer gene therapy

Completion of the human genome sequencing in 2001 has been regarded as a major milestone in terms of understanding the underlying mechanisms that contribute to the development of diseases. Cancer is one of the primary causes of death in the general population and is highly associated with the aberrant expression of genes that modulate cell proliferation, cell cycle, angiogenesis, and metastasis. Hence, target-specific gene therapy has become an essential approach to treat cancer. RNA interference (RNAi) is a strategy designed to selectively target and silence ‘druggable’ and ‘undruggable’ genes with less toxicity and greater specificity than other conventional treatments including chemotherapy. In this chapter, we will provide insights about the techniques of RNA delivery for cancer gene therapy as well as what to consider from the standpoint of targeting and silencing efficacy, stability, and safety.