Biomedical Engineering Reference
In-Depth Information
Chapter 6
Monika Dominska and Derek M. Dykxhoorn
John T. Macdonald Foundation Department of Human Genetics and the
Department of Microbiology and Immunology, University of Miami Miller
School of Medicine, 1501 NW 10th Ave, Miami, FL 33136, USA
DDykxhoorn@med.miami.edu
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.
6.1 Introduction
RNA interference (RNAi) is a highly conserved regulatory mechanism
of post-transcriptional gene silencing that uses small double-
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