Author(s): Zhu N, Liggitt D, Liu Y, Debs R
Abstract Share this page
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.
This article was published in Science
and referenced in Journal of Genetic Syndromes & Gene Therapy
- Ruben Artero
A Drosophila high-throughput drug screening platform identifies inhibitors of misregulated alternative splicing events in myotonic dystrophy
PPT Version | PDF Version