共 166 条
The design and exogenous delivery of siRNA for post-transcriptional gene silencing
被引:67
作者:
Gilmore, IR
Fox, SP
Hollins, AJ
Muhammad, SB
Akhtar, S
机构:
[1] Cardiff Univ, Welsh Sch Pharm, Ctr Genome Based Therapeut, Cardiff CF10 3XF, S Glam, Wales
[2] Univ Oxford, Dept Biochem, Oxford OX1 3Q, England
关键词:
small interfering RNA;
RNAi;
design;
delivery;
vectors;
liposomes;
dendrimer;
polymer;
D O I:
10.1080/10611860400006257
中图分类号:
R9 [药学];
学科分类号:
1007 ;
摘要:
RNA interference (RNAi) is a natural cellular process that effects post-transcriptional gene silencing in eukaryotic systems. Small interfering RNA (siRNA) molecules are the key intermediaries in this process which when exogenously administered can inhibit or "silence" the expression of any given target gene. Thus, siRNA molecules hold great promise as biological tools and as potential therapeutic agents for targeted inhibition of disease-causing genes. However, key challenges to the effective and widespread use of these polyanionic, macromolecular duplexes of RNA are their appropriate design and efficient delivery to cells in vitro and in vivo. This review highlights the current strategies used in the design of effective siRNA molecules and also summarises the main strategies being considered for the exogenous delivery of siRNA for both in vitro and in vivo applications.
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页码:315 / 340
页数:26
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