High potency silencing by single-stranded boranophosphate siRNA.

Journal Article (Journal Article)

In RNA interference (RNAi), double-stranded short interfering RNA (ds-siRNA) inhibits expression from complementary mRNAs. Recently, it was demonstrated that short, single-stranded antisense RNA (ss-siRNA) can also induce RNAi. While ss-siRNA may offer several advantages in both clinical and research applications, its overall poor activity compared with ds-siRNA has prevented its widespread use. In contrast to the poor gene silencing activity of native ss-siRNA, we found that the silencing activity of boranophosphate-modified ss-siRNA is comparable with that of unmodified ds-siRNA. Boranophosphate ss-siRNA has excellent maximum silencing activity and is highly effective at low concentrations. The silencing activity of boranophosphate ss-siRNA is also durable, with significant silencing up to 1 week after transfection. Thus, we have demonstrated that boranophosphate-modified ss-siRNA can silence gene expression as well as native ds-siRNA, suggesting that boranophosphate-modified ss-siRNAs should be investigated as a potential new class of therapeutic agents.

Full Text

Duke Authors

Cited Authors

  • Hall, AHS; Wan, J; Spesock, A; Sergueeva, Z; Shaw, BR; Alexander, KA

Published Date

  • 2006

Published In

Volume / Issue

  • 34 / 9

Start / End Page

  • 2773 - 2781

PubMed ID

  • 16717282

Pubmed Central ID

  • PMC1464415

Electronic International Standard Serial Number (EISSN)

  • 1362-4962

Digital Object Identifier (DOI)

  • 10.1093/nar/gkl339


  • eng

Conference Location

  • England