Biology Reference
In-Depth Information
Chapter 6
Nanomedicines for Systemic Delivery
of RNAi Therapeutics
Dan Peer
Abstract
The discovery of RNA interference (RNAi), a ubiquitous cellular pathway
of gene regulation that is dysregulated in many types of diseases, provides an
exciting opportunity for relatively rapid and revolutionary approaches to drug
design. Small RNAs that harness the RNAi machinery may become the next class
of drugs for treating a variety of diseases. However, targeting of RNAi molecules
into specific tissues and cells is still a hurdle. Major attempts are being made to
develop carriers that could overcome systemic and cellular barriers. This chapter
will present the recent progress in this emerging field, focusing on strategies for
systemic cellular targeting using different types of nano-sized vectors. Selective
cellular targeting of RNAi is considered as the major hurdle translating this new
class of drugs into clinical practice.
6.1
Introduction
RNA interference (RNAi) is a natural cellular mechanism for RNA-guided regula-
tion of gene expression. This regulation is carried out by double-stranded ribonu-
cleic acid (dsRNA) that suppress the expression of speci fi c genes with complementary
nucleotide sequences either by degrading specific messenger RNA (mRNA) or by
blocking mRNA translation. RNAi can be activated by expressing short hairpin
RNA (shRNA) or by introducing synthetic small interfering RNAs (siRNAs) directly
into the cell cytoplasm with viral and nonviral vectors [
1,
2
] .
D. Peer (
*
)
Laboratory of Nanomedicine, Department of Cell Research and Immunology,
George S. Wise Faculty of Life Science, Tel Aviv University, Tel Aviv, Israel
Center for Nanoscience and Nanotechnology , Tel Aviv University , Tel Aviv , Israel
e-mail: peer@tauex.tau.ac.il
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