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Chapter 10
Aptamer-Mediated siRNA Targeting
Jiehua Zhou and John J. Rossi
Abstract
RNA interference (RNAi) is a sequence-specific mechanism for post-
transcriptional inhibition of gene expression. As such, it is an attractive approach
for the therapeutic treatment of a wide variety of human maladies. Although con-
ceptually elegant, there are key barriers to the widespread clinical application of this
process. One of the most formidable impediments to clinical translation of RNAi is
safe and effective delivery of the siRNAs to the desired target tissue at therapeutic
doses. In this regard, the advent of versatile aptamer technology has prompted the
development of aptamer-mediated cell-type-specific delivery for targeted RNAi
triggers. In this chapter, we explore the developments of cell-type-specific aptamer
applications. We also highlight recent advances of aptamers as functionalized nano-
carriers for targeted siRNA delivery.
10.1
Introduction
Small interfering RNAs (siRNAs) are 21-22-base-long RNAs that guide the
sequence-specific degradation of target mRNAs [
1,
2
] . The essential feature of
the RNAi mechanism is the exquisite sequence specificity, which derives from
J. Zhou
Division of Molecular and Cellular Biology, Beckman Research Institute
of City of Hope, City of Hope , Duarte , CA , USA
J. J. Rossi (
*
)
Irell and Manella Graduate School of Biological Sciences, Division of Molecular
and Cellular Biology, Beckman Research Institute of City of Hope, City of Hope,
Duarte, CA, USA
e-mail: jrossi@coh.org
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