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PMID: 20226078 Published · epublish English Journal Article

Aptamer-targeted cell-specific RNA interference.

Silence ·Vol. 1 ·No. 1 ·2010-02-01 ·Pages 4

Zhou J, Rossi JJ

Abstract

This potent ability of small interfering (si)RNAs to inhibit the expression of complementary RNA transcripts is being exploited as a new class of therapeutics for a variety of diseases. However, the efficient and safe delivery of siRNAs into specific cell populations is still the principal challenge in the clinical development of RNAi therapeutics. With the increasing enthusiasm for developing targeted delivery vehicles, nucleic acid-based aptamers targeting cell surface proteins are being explored as promising delivery vehicles to target a distinct disease or tissue in a cell-type-specific manner. The aptamer-based delivery of siRNAs can often enhance the therapeutic efficacy and reduce the unwanted off-target effects of siRNAs. In particular, for RNA interference-based therapeutics, aptamers represent an efficient agent for cell type-specific, systemic delivery of these oligonucleotides. In this review, we summarize recent attractive developments in creatively using cell-internalizing aptamers to deliver siRNAs to target cells. The optimization and improvement of aptamer-targeted siRNAs for clinical translation are further highlighted.

Authors & Affiliations
2 authors, click to expand affiliations / ORCID
Zhou Jiehua
Division of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, City of Hope, Duarte, CA 91010, USA. jrossi@coh.org.
Rossi John J
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Article Info
Journal
Silence
Abbr.
Silence
ISSN
1758-907X
DOI
10.1186/1758-907X-1-4
Email
jrossi@coh.org
Published
2010-02-01
Epub
2010-00-01
Pages
4
Language
English
Region
England
NLM ID
101525809
PMCID
PMC2835998
Grants
NHLBI NIH HHS · R01 HL074704 · United States
External Links
PubMed source DOI: 10.1186/1758-907X-1-4 PMC Full Text
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