- 1、本文档共16页,可阅读全部内容。
- 2、原创力文档(book118)网站文档一经付费(服务费),不意味着购买了该文档的版权,仅供个人/单位学习、研究之用,不得用于商业用途,未经授权,严禁复制、发行、汇编、翻译或者网络传播等,侵权必究。
- 3、本站所有内容均由合作方或网友上传,本站不对文档的完整性、权威性及其观点立场正确性做任何保证或承诺!文档内容仅供研究参考,付费前请自行鉴别。如您付费,意味着您自己接受本站规则且自行承担风险,本站不退款、不进行额外附加服务;查看《如何避免下载的几个坑》。如果您已付费下载过本站文档,您可以点击 这里二次下载。
- 4、如文档侵犯商业秘密、侵犯著作权、侵犯人身权等,请点击“版权申诉”(推荐),也可以打举报电话:400-050-0827(电话支持时间:9:00-18:30)。
查看更多
celltype-specificdeliveryofsirnaswithaptamer-sirnachimeras
Cell type-specific delivery of siRNAs
with aptamer-siRNA chimeras
Sullenger, B. A. et al
Duke Center for Translational Research,
Duke University
Nature Biotechnology, 2006, 24, 1005
Julia Vargas
November 25th, 2006
Julia Vargas @ Wipf Group 1 11/25/2006Discovery of RNAi
( 2006 Nobel Prize in Physiology or Medicine
“for their discovery of RNA interference –
gene silencing by double-stranded RNA”
Andrew Z. Fire
Stanford University Craig C. Mello
University of
Massachusetts Nobel Prize: physiology or medicine, reverse side. [Photograph]. Retrieved November 12, 2006, from Encyclopedia Britannica Online:
/eb/art-18048
Julia Vargas @ Wipf Group 2 11/25/2006Discovery of RNAi
(Regulation of gene expression in C. Elegans
? dsRNA induces highly
specific gene silencing
? Silencing triggers:
- exogenously induced dsRNA,
RNA viruses, transposons,
endogenous short dsRNAs
? Posttranscriptional
cytoplasmic mechanism
? Systemic Silencing
? Effects first generation
progeny
/nobel_prizes/medicine/laureates/2006/adv.html
Julia Vargas @ Wipf Group 3 11/25/2006RNA interference
? dsRNA when injected into cell is cleaved by Dicer
- A Class 3 RNase III endonuclease
Structural Features:
? 21-25 nt sequence, composed of duplex (19-23 bp)
? 3’ overhang, 2-nt segment
? 5’-phosphate and 3’-hydroxyl termini
Nat. Struct. Biol. 3, 214 (2004).
Julia Vargas @ Wipf Group 4 11/25/2006Mechanistic Model
? dsRNA recognized by Dicer
? Endonucleolytic cleavage
? siRNA-RISC complex
targets homologous mRNAs
Nature. 418, 244 (2002)
Julia Vargas @ Wipf Group 5 11/25/2006RNAi Therapeutics
Advantages:
? Target-gene specificity
? Low immunogenicity
? Simplicity of design/testing
- easily synthesized, large scale
- low cost
? Amenable to chemical
modifications
? Size: aptamer vs antibody
15 kDa 150 kDa
CEN. 2006, 84(46), 16-23
Julia Vargas @ Wipf Group 6 11/25/2006Cell type-specific delivery of siRNAs
? RNA-based approach for specific delivery of siRNA
? Exploit structural potential of RNA to
您可能关注的文档
- bd6cr4王忠华bd8te2010.4.4@chinahamexpo2010.doc
- bergencommunitycollege.doc
- blackberry隐私政策.doc
- beijing-fanucpmc.doc
- brotheriprint&scan)(windowsphone描说明书(适用于.doc
- bsspn}ns×c§.doc
- cacti中文版安装配置-chinaunix博客.doc
- cambridgeinternationalexaminationscambridgeinternational.doc
- carbon――guideforauthors(作者指南.doc
- categorizingemotionalvocabulariesinchinesenatural.doc
文档评论(0)