Systemic Gene Expression After Intravenous DNA Delivery into Adult Mice

TLDR

The study aims to develop a method for direct gene transfer into adult mice that enables generalized or tissue‑specific expression for rapid analysis of transgene effects. A single intravenous injection of plasmid–cationic liposome complexes transfected virtually all tissues in adult mice, with sustained expression for at least nine weeks, no toxicity, and tissue‑specific targeting achievable via promoter choice.

Abstract

Direct gene transfer into adult animals resulting in generalized or tissue-specific expression would facilitate rapid analysis of transgene effects and allow precise in vivo manipulation of biologic processes at the molecular level. A single intravenous injection of expression plasmid:cationic liposome complexes into adult mice efficiently transfected virtually all tissues. In addition to vascular endothelial cells, most of the extravascular parenchymal cells present in many tissues including the lung, spleen, lymph nodes, and bone marrow expressed the transgene without any apparent treatment-related toxicity. The transgene was still expressed in large numbers of cells in multiple tissues for at least 9 weeks after a single injection. Expression could be targeted to specific tissues and cell types, depending on the promoter element used.