Special delivery: Targeted therapy with small RNAs

D. Peer; J. Lieberman

doi:10.1038/gt.2011.56

Special delivery: Targeted therapy with small RNAs

D. Peer, J. Lieberman

Research output: Contribution to journal › Review article › peer-review

121 Scopus citations

Abstract

Harnessing RNA interference using small RNA-based drugs has great potential to develop drugs designed to knock down expression of any disease-causing gene, thereby greatly expanding the universe of possible drug targets. However, delivering small RNAs into specific tissues and cells is still a hurdle. Here, we review recent progress in overcoming systemic, local and cellular barriers to RNA drug delivery, focusing on strategies for targeted uptake.

Original language	English (US)
Pages (from-to)	1127-1133
Number of pages	7
Journal	Gene Therapy
Volume	18
Issue number	12
DOIs	https://doi.org/10.1038/gt.2011.56
State	Published - Dec 2011

Keywords

aptamer
drug
liposome
nanoparticle
siRNA

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics

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10.1038/gt.2011.56

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title = "Special delivery: Targeted therapy with small RNAs",

abstract = "Harnessing RNA interference using small RNA-based drugs has great potential to develop drugs designed to knock down expression of any disease-causing gene, thereby greatly expanding the universe of possible drug targets. However, delivering small RNAs into specific tissues and cells is still a hurdle. Here, we review recent progress in overcoming systemic, local and cellular barriers to RNA drug delivery, focusing on strategies for targeted uptake.",

keywords = "aptamer, drug, liposome, nanoparticle, siRNA",

author = "D. Peer and J. Lieberman",

note = "Funding Information: This work was supported, in part, by grants from the Alon Foundation, the Marie Curie IRG-FP7 of the European Union, Levy Family Trust and ISF (181/10) to DP, the Breast Cancer Research Program of the US Department of Defense to JL, and by a joint BSF grant (2009107) to DP and JL.",

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N2 - Harnessing RNA interference using small RNA-based drugs has great potential to develop drugs designed to knock down expression of any disease-causing gene, thereby greatly expanding the universe of possible drug targets. However, delivering small RNAs into specific tissues and cells is still a hurdle. Here, we review recent progress in overcoming systemic, local and cellular barriers to RNA drug delivery, focusing on strategies for targeted uptake.

AB - Harnessing RNA interference using small RNA-based drugs has great potential to develop drugs designed to knock down expression of any disease-causing gene, thereby greatly expanding the universe of possible drug targets. However, delivering small RNAs into specific tissues and cells is still a hurdle. Here, we review recent progress in overcoming systemic, local and cellular barriers to RNA drug delivery, focusing on strategies for targeted uptake.

KW - aptamer

KW - drug

KW - liposome

KW - nanoparticle

KW - siRNA

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Special delivery: Targeted therapy with small RNAs

Abstract

Keywords

ASJC Scopus subject areas

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