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Nature Methods - 3, 689 - 695 (2006)
Published online: 23 August 2006; | doi:10.1038/nmeth927

Expressing short hairpin RNAs in vivo

Ola Snøve Jr1, 2, 3 & John J Rossi1

1  Division of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, California 91010, USA.

2  Interagon AS, Laboratoriesenteret, NO-7006 Trondheim, Norway.

3  Department of Cancer Research and Molecular Medicine, Faculty of Medicine, Norwegian University of Science and Technology, NO-7006 Trondheim, Norway.

Correspondence should be addressed to John J Rossi jrossi@bricoh.edu

Promoter-based expression of short hairpin RNAs (shRNAs) may in principle provide stable silencing of genes in any tissue. As for all approaches that require transgene expression, safe delivery is the biggest obstacle, but toxicity can also occur via expression of the sequence itself. Innate immunity mechanisms can be triggered by expressed hairpin RNAs, critical cellular factors can be saturated, and genes other than the intended target can be silenced. Nevertheless, shRNAs constitute a valuable tool for in vivo research and have great therapeutic potential if the challenges with delivery and side effects are appropriately addressed.

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Nature Methods
ISSN: 1548-7091
EISSN: 1548-7105
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