Biomedical Engineering Reference
In-Depth Information
Chapter 5
Cell-Specific Aptamer-Functionalized
RNAi: A New Prospect for Targeted
siRNA Delivery
Jiehua Zhou and John J. Rossi
*
Molecular and Cellular Biology, Beckman Research Institute of City of Hope,
1500 E Duarte Road, Duarte, CA 91010, USA
*
jrossi@coh.org
In recent years, nucleic acid based therapeutics has progressed
rapidly. In addition to their utility as a stand-alone strategy, these
therapeutic nucleic acids that include small interfering (si)RNAs,
microRNAs, aptamers, antisense DNAs, DNAzymes, and ribozymes
can potentially be used as adjuvants for multipronged drug treatment.
In particular, the highly specific mechanism of RNA interference
(RNAi) which inhibits the expression of disease genes is extremely
attractive for the treatment of a wide variety of human maladies.
However, one of the most formidable impediments to clinical
translation of RNAi is effective delivery of the small interfering RNAs
(siRNAs) to specific cells or tissues at therapeutic doses. Currently,
extensive efforts to develop various internalizing molecules have
been made for target-specific siRNA delivery. Among them, cell-
specific aptamers represent an exciting approach for targeted siRNA
delivery. In this chapter, we review the current advances of cell-
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