4.5 Review

Special delivery: targeted therapy with small RNAs

Journal

GENE THERAPY
Volume 18, Issue 12, Pages 1127-1133

Publisher

NATURE PUBLISHING GROUP
DOI: 10.1038/gt.2011.56

Keywords

siRNA; drug; liposome; nanoparticle; aptamer

Funding

  1. Alon Foundation
  2. European Union
  3. Levy Family Trust
  4. ISF [181/10]
  5. US Department of Defense
  6. BSF [2009107]

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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. Gene Therapy (2011) 18, 1127-1133; doi:10.1038/gt.2011.56; published online 14 April 2011

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