CELL-TYPE SPECIFIC SYSTEMIC DELIVERY OF siRNA
Inventors: Irwin Song, Judy Lieberman
Invention Types: Therapeutics, Research Tool
Research Areas: Oncology/Hematology
Keywords: For More Information Contact: Dietz, Ryan
The effectiveness of gene silencing using RNAi is dependent on efficient delivery of siRNAs into cells. This
delivery method allows specific disease causing genes to be turned off by siRNAs that enter only specific cells
in both in vitro and in vivo settings, thereby avoiding potential side effects of delivery into non-targeted cells.
Through cell specific systemic delivery of siRNAs, we have taken a significant step towards using siRNAs as
human therapeutics. |
This delivery method utilizes a cell-targeting moiety that binds to a specific molecule on a target cell such as a
cell-surface receptor or surface antigen and an RNA interference-binding moiety that binds to the siRNA of
interest. Experiments have shown that this delivery method selectively delivers the siRNA only into the cells
expressing a specific surface receptors or antigen. The efficacy has been demonstrated in vivo using both
subcutaneous and intravenous administration. In one experiment, the tagged siRNAs were absorbed only by
mouse melanoma cells engineered to carry a specific receptor and not by normal melanoma cells. Also, siRNAs
designed to shut down known cancer-causing genes were injected into mice implanted with engineered
melanoma cells. Results after nine days showed that the tumors were about half the weight of tumors in the
- Delivery of small interfering siRNAs to specific cell types for human therapeutics.
- Self-delivering reagents as a more effective alternative to electroporation, chemical, lipid or polymer-based transfection methods.
Nonexclusive worldwide licenses for therapeutics and research reagents
Key Publications: Nature Biotechnology 23: 709-717, 2005
Proc Natl Acad Sci U S A. 2007 Mar 6;104(10):4095-100. Epub 2007 Feb 28.