Gabriel Lopez-Berestein

RNAi: A New Paradigm in Cancer Gene Therapy

RNA interference (RNAi) has revolutionized the field of gene therapy and opened up new opportunities for personalized treatments. However, several challenges remain for gene therapy. Therefore, new approaches for gene regulatory therapies are needed to overcome these challenges. In this chapter, we discuss the clinical significance of the RNAi machinery, clinical applications, delivery systems, off-target effects, imaging, and clinical trials. The remarkable advances in the design, delivery, and understanding of RNAi-based therapeutics predict a bright future for their development as therapeutic agents. It is well established that once a gene is identified as an important player in tumor progression or metastasis, siRNA is a feasible alternative to modulate its expression. Moreover, the development of new delivery systems will further advance the efficiency and localization of siRNA delivery to specific tissues and organs. Concurrently, the development of “intelligent probes” to identify siRNA function in addition to localization will further advance the evaluation of new formulations using imaging techniques.