ABSTRACT
RNA interference (RNAi)-based gene-silencing technologies provide a novel approach for the treatment of a variety of diseases through the sequence-specific silencing of gene expression. The application of small interfering RNA (siRNA) as potential therapeutic agents requires the development of clinically feasible delivery strategies that enhance their pharmacological properties. To be eff ective, siRNAs must be delivered to and taken up by specific target cells and tissues, enter the cytoplasm, and associate with the RNA-induced silencing complex (RISC) to guide the sequence-specific cleavage of appropriate messenger RNA (mRNA). This chapter will focus on recent progress made in the development of safe and eff ective therapeutic strategies for the siRNA-based silencing of gene expression.
