Lipid-modified aminoglycoside derivatives for in vivo siRNA delivery
Yunlong Zhang1, Jeisa M Pelet, Daniel A Heller
1David H. Koch Institute for Integrative, Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Anesthesiology, Children's Hospital Boston, Boston, MA 02115, USA.
Abstract:
Rationally designed siRNA delivery materials that are enabled by lipid-modified aminoglycosides are demonstrated. Leading materials identified are able to self-assemble with siRNA into well-defined nanoparticles and induce efficient gene knockdown both in vitro and in vivo. Histology studies and liver function tests reveal that no apparent toxicity is caused by these nanoparticles at doses over two orders of magnitude.
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