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Novel class of amphipathic DNA-based transporters for convenient cellular delivery of uncharged or negatively charged nucleic acid sequences

Friday, September 18, 2015 — Poster Session IV

12:00 p.m. – 1:30 p.m.
FAES Terrace


  • HV Jain
  • D Verthelyi
  • SL Beaucage


A simple and convenient method for the delivery of uncharged peptide nucleic acids (PNA) or phosphorodiamidate morpholino (PMO) oligomers into mammalian cells is presented; the procedure consists of extending these nucleic acid sequences with a short (6-mer) PNA-polyA or PMO-polyA tail. Recognition of the polyA-tailed PNA or PMO oligomers by an 8-mer amphipathic trans-acting polythymidylic thiophosphate triester DNA element (dTtaPS) results in efficient internalization of these oligomers into various cell lines. Furthermore, the DNA-based transporters reported herein were tailored to effectively deliver negatively charged nucleic acids such as DNA plasmids or siRNAs in various mammalian cell lines. Data conclusively show that amphipathic DNA-based transporters represent a novel class of cellular transporters for efficient delivery of negatively charged and uncharged polyA-tailed nucleic acid sequences in live mammalian cells.

Category: Biomedical Engineering and Biophysics