Yunlong Zhang, Jeisa M. Pelet, Daniel A. Heller, Yizhou Dong, Delai Chen, Zhen Gu, Brian J. Joseph, Jasmine Wallas and Daniel G. Anderson Lipid-Modified Aminoglycoside Derivatives for In Vivo siRNA Delivery Advanced Materials 25
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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