Systemic siRNA delivery via hydrodynamic intravascular injection

被引:49
作者
Lewis, David L.
Wolff, Jon A.
机构
[1] Mirus Bio Corp, Madison, WI 53719 USA
[2] Univ Wisconsin, Sch Med, Waisman Ctr, Madison, WI 53705 USA
关键词
siRNA; hydrodynamic injection; gene knockdown;
D O I
10.1016/j.addr.2007.03.002
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
The main barrier to the use of RNAi in mammalian systems is the difficulty in delivering siRNA or shRNA to the appropriate tissues. Although progress has been made in this area, many of the technologies developed require specialized expertise and reagents that are beyond the reach of most investigators. In contrast, the hydrodynamic injection technique is simple to perform and enables highly efficient delivery of naked, unmodified siRNA to a number of tissues, especially the liver. This review describes the development of the technique and explores the possible mechanisms that enable uptake of siRNA to biological effect. Examples of the use of hydrodynamic injection in animal models of disease and for the study of gene function are presented and discussed. (C) 2007 Elsevier B.V. All rights reserved.
引用
收藏
页码:115 / 123
页数:9
相关论文
共 69 条
[1]   Hydrodynamics-based transfection of the liver: entrance into hepatocytes of DNA that causes expression takes place very early after injection [J].
Andrianaivo, F ;
Lecocq, M ;
De Coninck, SW ;
Wattiaux, R ;
Jadot, M .
JOURNAL OF GENE MEDICINE, 2004, 6 (08) :877-883
[2]   Insights into the kinetics of siRNA-mediated gene silencing from live-cell and live-animal bioluminescent imaging [J].
Bartlett, DW ;
Davis, ME .
NUCLEIC ACIDS RESEARCH, 2006, 34 (01) :322-333
[3]   Short 5′-phosphorylated double-stranded RNAs induce RNA interference in Drosophila [J].
Boutla, A ;
Delidakis, C ;
Livadaras, I ;
Tsagris, M ;
Tabler, M .
CURRENT BIOLOGY, 2001, 11 (22) :1776-1780
[4]   Gene silencing in the endocrine pancreas mediated by short-interfering RNA [J].
Bradley, SP ;
Rastellini, C ;
da Costa, MA ;
Kowalik, TF ;
Bloomenthal, AB ;
Brown, M ;
Cicalese, L ;
Basadonna, GP ;
Uknis, ME .
PANCREAS, 2005, 31 (04) :373-379
[5]   The efficient expression of intravascularly delivered DNA in rat muscle [J].
Budker, V ;
Zhang, G ;
Danko, I ;
Williams, P ;
Wolff, J .
GENE THERAPY, 1998, 5 (02) :272-276
[6]  
Budker V, 1996, GENE THER, V3, P593
[7]  
Budker V, 2000, J GENE MED, V2, P76, DOI 10.1002/(SICI)1521-2254(200003/04)2:2<76::AID-JGM97>3.0.CO
[8]  
2-4
[9]   Mechanism of plasmid delivery by hydrodynamic tail vein injection.: II.: Morphological studies [J].
Budker, Vladimir G. ;
Subbotin, Vladimir M. ;
Budker, Tatiana ;
Sebestyen, Magdolna G. ;
Zhang, Guofeng ;
Wolff, Jon A. .
JOURNAL OF GENE MEDICINE, 2006, 8 (07) :874-888
[10]   Transient siRNA-mediated attenuation of liver expression from an α-galactosidase a plasmid reduces subsequent humoral immune responses to the transgene product in mice [J].
Chu, QM ;
Joseph, M ;
Przybylska, M ;
Yew, NS ;
Scheule, RK .
MOLECULAR THERAPY, 2005, 12 (02) :264-273