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Lipid-modified aminoglycoside derivatives for in vivo siRNA delivery.

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.

SUBMITTER: Zhang Y 

PROVIDER: S-EPMC3898629 | biostudies-literature | 2013 Sep

REPOSITORIES: biostudies-literature

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Lipid-modified aminoglycoside derivatives for in vivo siRNA delivery.

Zhang Yunlong Y   Pelet Jeisa M JM   Heller Daniel A DA   Dong Yizhou Y   Chen Delai D   Gu Zhen Z   Joseph Brian J BJ   Wallas Jasmine J   Anderson Daniel G DG  

Advanced materials (Deerfield Beach, Fla.) 20130627 33

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. ...[more]

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