A peptidomimetic siRNA transfection reagent for highly effective gene silencing

Yeliz Utku, Elinor Dehan, Ouathek Ouerfelli, Fabio Piano, Ronald N. Zuckermann, Michele Pagano, Kent Kirshenbaum

Research output: Contribution to journalArticle

Abstract

RNA interference (RNAi) techniques hold forth great promise for therapeutic silencing of deleterious genes. However, clinical applications of RNAi require the development of safe and efficient methods for intracellular delivery of small interfering RNA (siRNA) oligonucleotides specific to targeted genes. We describe the use of a lipitoid, a cationic oligopeptoid-phospholipid conjugate, for non-viral transfection of synthetic siRNA oligos in cell culture. This peptidomimetic delivery vehicle allows for efficient siRNA transfection in a variety of human cell lines with negligible toxicity and promotes extensive downregulation of the targeted genes at both the protein and the mRNA level. We compare the lipitoid reagent to a standard commercial transfection reagent. The lipitoid is highly efficient even in primary IMR-90 human lung fibroblasts in which other commercial reagents are typically ineffective.

Original languageEnglish (US)
Pages (from-to)312-317
Number of pages6
JournalMolecular BioSystems
Volume2
Issue number6
DOIs
StatePublished - 2006

ASJC Scopus subject areas

  • Biotechnology
  • Molecular Biology

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