Targeted delivery of antisense oligonucleotides in cancer

F. Pastorino, D. Stuart, M. Ponzoni, T. M. Allen

Research output: Contribution to journalArticlepeer-review


Formulations of antisense oligonucleotides (asODNs) against c-myb or c-myc protooncogenes have been prepared by a new technique that sequesters cationic lipid in the interior of a lipid particle. This technique results in high loading efficiency for the asODNs, small particle size and good stability. When targeted against melanoma cells or neuroblastoma cells via anti-GD2 coupled at the particle surface, increased cell binding to the cells could be demonstrated. Targeted formulations showed greater inhibition of cell proliferation compared to non-targeted formulations or free drug. Inhibition of cell proliferation was demonstrated to be due to down-regulation of c-myb or c-myc protein expression. The formulations have long-circulation times in vivo, and evaluation for in vivo antitumor activity is currently underway.

Original languageEnglish
Pages (from-to)69-75
Number of pages7
JournalJournal of Controlled Release
Issue number1-3
Publication statusPublished - Jul 6 2001


  • Anti-GD
  • Antisense oligonucleotides
  • Liposomes
  • Melanoma
  • Targeted drug delivery

ASJC Scopus subject areas

  • Pharmaceutical Science


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