Biodistribution of phosphodiester and phosphorothioate siRNA

Dwaine A. Braasch, Zain Paroo, Anca Constantinescu, Gang Ren, Orhan K. Öz, Ralph P. Mason, David R. Corey

Research output: Contribution to journalArticlepeer-review

219 Scopus citations

Abstract

Short interfering RNAs (siRNAs) are valuable tools for analyzing protein function in mammalian cell culture. This success has led to high expectations for in vivo and therapeutic applications. However, the pharmacokinetic properties of siRNA are not known. Here we report the biodistribution of a phosphodiester (PO) siRNA duplex and examine the effect of phosphorothioate (PS) linkages. Our findings indicate that biodistribution of siRNA is similar to that for single-stranded antisense oligonucleotides and offer insights for use of siRNA in vivo.

Original languageEnglish (US)
Pages (from-to)1139-1143
Number of pages5
JournalBioorganic and Medicinal Chemistry Letters
Volume14
Issue number5
DOIs
StatePublished - Mar 8 2004

Keywords

  • Biodistribution
  • Phosphorothioate
  • Planar imaging
  • siRNA

ASJC Scopus subject areas

  • Biochemistry
  • Molecular Medicine
  • Molecular Biology
  • Pharmaceutical Science
  • Drug Discovery
  • Clinical Biochemistry
  • Organic Chemistry

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