Lipid-Modified Aminoglycoside Derivatives for In Vivo siRNA Delivery

被引：34

作者：

Zhang, Yunlong ^{[1
,2
,3
]}

Pelet, Jeisa M. ^{[1
,3
]}

Heller, Daniel A. ^{[2
,5
]}

Dong, Yizhou ^{[3
]}

Chen, Delai ^{[1
]}

Gu, Zhen ^{[1
,3
]}

Joseph, Brian J. ^{[2
,5
]}

Wallas, Jasmine ^{[5
]}

Anderson, Daniel G. ^{[1
,2
,3
,4
]}

机构：

[1] MIT, David H Koch Inst Integrat Canc Res, Cambridge, MA 02139 USA

[2] MIT, Dept Chem Engn, Cambridge, MA 02139 USA

[3] Childrens Hosp Boston, Dept Anesthesiol, Boston, MA 02115 USA

[4] MIT, Inst Med Engn & Sci, Cambridge, MA 02139 USA

[5] Mem Sloan Kettering Canc Ctr, Mol Pharmacol & Chem Program, New York, NY 10065 USA

来源：

ADVANCED MATERIALS | 2013年 / 25卷 / 33期

关键词：

siRNA; drug delivery; nanoparticle; gene knockdown; aminoglycoside; DOUBLE-STRANDED-RNA; CATIONIC LIPIDS; NANOPARTICLES; INTERFERENCE; EFFICIENT; POTENT; VECTORS;

D O I：

10.1002/adma.201301917

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

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. Copyright © 2013 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

引用

页码：4641 / 4645

页数：5

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