Lipid-Modified Aminoglycoside Derivatives for In Vivo siRNA Delivery

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 liv...

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Bibliographic Details
Main Authors: Zhang, Yunlong, Heller, Daniel A., Dong, Yizhou, Chen, Delai, Gu, Zhen, Joseph, Brian J., Wallas, Jasmine, Anderson, Daniel Griffith, Pelet, Jeisa
Other Authors: Massachusetts Institute of Technology. Department of Chemical Engineering
Format: Article
Language:en_US
Published: Wiley-VCH Verlag GmbH & Co. 2014
Online Access:http://hdl.handle.net/1721.1/91256
https://orcid.org/0000-0001-5629-4798
Description
Summary: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.