Design and assembly of new nonviral RNAi delivery agents by microwave-assisted quaternization (MAQ) of tertiary amines

Bioconjug Chem. 2010 Sep 15;21(9):1581-7. doi: 10.1021/bc900482r.

Abstract

RNA interference (RNAi) is a gene-silencing phenomenon whereby double-stranded RNA (dsRNA) triggers the sequence-specific degradation of homologous mRNA. RNAi has been quickly and widely applied to discover gene functions and holds great potential to provide a new class of therapeutic agents. However, new chemistry and delivery approaches are greatly needed to silence disease-causing genes without toxic effects. We reasoned that conjugation of the cholesterol moiety to cationic lipids would enhance RNAi efficiencies and lower the toxic effects of lipid-mediated RNAi delivery. Here, we report the first design and synthesis of new cholesterol-conjugated cationic lipids for RNAi delivery using microwave-assisted quaternization (MAQ) of tertiary amines. This strategy can be employed to develop new classes of nonviral gene delivery agents under safe and fast reaction conditions.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Amines / chemistry*
  • Cations
  • Cholesterol / chemistry
  • Drug Delivery Systems / methods*
  • Drug Design*
  • Lipids / chemical synthesis*
  • Lipids / chemistry
  • Microwaves*
  • RNA Interference*
  • RNA, Double-Stranded / genetics
  • RNA, Double-Stranded / metabolism
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / metabolism

Substances

  • Amines
  • Cations
  • Lipids
  • RNA, Double-Stranded
  • RNA, Messenger
  • RNA, Small Interfering
  • Cholesterol