Nanotechnology for In vivo Targeted siRNA Delivery

被引：30
|
作者：
Dahlman, James E. [1 ,2 ,4 ]
Kauffman, Kevin J. [3 ,4 ]
Langer, Robert [1 ,2 ,3 ,4 ]
Anderson, Daniel G. [1 ,2 ,3 ,4 ]
机构：
[1] MIT, Inst Med Engn & Sci, Cambridge, MA 02139 USA
[2] MIT, Harvard MIT Div Hlth Sci & Technol, Cambridge, MA 02139 USA
[3] MIT, Dept Chem Engn, Cambridge, MA 02139 USA
[4] MIT, David H Koch Inst Integrat Canc Res, Cambridge, MA 02139 USA
来源：
NONVIRAL VECTORS FOR GENE THERAPY LIPID- AND POLYMER-BASED GENE TRANSFER | 2014年 / 88卷
关键词：
LIPID-LIKE MATERIALS; RNAI THERAPEUTICS; SYSTEMIC DELIVERY; DRUG-DELIVERY; ASIALOGLYCOPROTEIN RECEPTOR; NANOPARTICLES; GENE; INTERFERENCE; POTENT; VITRO;
D O I：
10.1016/B978-0-12-800148-6.00003-1
中图分类号：
Q3 [遗传学];
学科分类号：
071007 ; 090102 ;
摘要：
Small interfering RNAs (siRNAs) can specifically inhibit gene expression. As a result, they have tremendous scientific and clinical potential. However, the use of these molecules in patients and animal models has been limited by challenges with delivery. Intracellular RNA delivery is difficult; it requires a system that protects the siRNA from degradative nucleases in the bloodstream, minimizes clearance by the reticuloendothelial system, maximizes delivery to the target tissue, and promotes entry into, and out of, an endocytic vesicle. Despite these barriers, recent data suggest that RNA may be targeted to cells of interest in vivo. Herein we outline strategies for targeted siRNA delivery, and describe how these strategies may be improved.
引用
收藏
页码：37 / 69
页数：33
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