Small-Molecule-Mediated Cleavage of RNA in Living Cells

被引：24

作者：

Guan, Lirui ^{[1
]}

Disney, Matthew D. ^{[1
]}

机构：

[1] Scripps Res Inst, Dept Chem, Jupiter, FL 33458 USA

来源：

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION | 2013年 / 52卷 / 05期

基金：

美国国家卫生研究院;

关键词：

bioorganic chemistry; chemical biology; nucleic acids; RNA; RNA recognition; MYOTONIC-DYSTROPHY TYPE-1; SECONDARY STRUCTURE; TARGETS; REPEAT; RIBOSWITCHES; TOXICITY; REVERSAL; DESIGN;

D O I：

10.1002/anie.201206888

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

Killing the message: An approach to direct the cleavage of RNA targets with small molecules in living cells is described (see scheme). A bifunctional small molecule (purple) that recognizes a specific three nucleotide repeat sequence and cleaves that sequence in response to light was shown to be effective at degrading the myotonic dystrophy type 1 (DM1) extended repeat RNAs, thereby affecting biological functions. Copyright © 2013 WILEY-VCH Verlag GmbH & Co. KGaA, Weinheim.

引用

收藏

页码：1462 / 1465

页数：4

相关论文

共 29 条

[1] Thioamide Hydroxypyrothiones Supersede Amide Hydroxypyrothiones in Potency against Anthrax Lethal Factor [J].

Agrawal, Arpita ;

de Oliveira, Cesar Augusto F. ;

Cheng, Yuhui ;

Jacobsen, Jennifer A. ;

McCammon, J. Andrew ;

Cohen, Seth M. .

JOURNAL OF MEDICINAL CHEMISTRY, 2009, 52 (04) :1063-1074

[2] Antibacterial lysine analogs that target lysine riboswitches [J].

Blount, Kenneth F. ;

Wang, Joy Xin ;

Lim, Jinsoo ;

Sudarsan, Narasimhan ;

Breaker, Ronald R. .

NATURE CHEMICAL BIOLOGY, 2007, 3 (01) :44-49

[3] Riboswitches as antibacterial drug targets [J].

Blount, Kenneth F. ;

Breaker, Ronald R. .

NATURE BIOTECHNOLOGY, 2006, 24 (12) :1558-1564

[4] Studies of RNA cleavage by photolysis of N-hydroxypyridine-2(1H)-thione.: A new photochemical footprinting method [J].

Chaulk, SG ;

Pezacki, JP ;

MacMillan, AM .

BIOCHEMISTRY, 2000, 39 (34) :10448-10453

[5] A small molecule microarray platform to select RNA internal loop-ligand interactions [J].

Childs-Disney, Jessica L. ;

Wu, Meilan ;

Pushechnikov, Alexei ;

Aminova, Olga ;

Disney, Matthew D. .

ACS CHEMICAL BIOLOGY, 2007, 2 (11) :745-754

[6] Rationally Designed Small Molecules Targeting the RNA That Causes Myotonic Dystrophy Type 1 Are Potently Bioactive [J].

Childs-Disney, Jessica L. ;

Hoskins, Jason ;

Rzuczek, Suzanne G. ;

Thornton, Charles A. ;

Disney, Matthew D. .

ACS CHEMICAL BIOLOGY, 2012, 7 (05) :856-862

[7] Progress in antisense technology [J].

Crooke, ST .

ANNUAL REVIEW OF MEDICINE, 2004, 55 :61-95

[8] RNA pathogenesis of the myotonic dystrophies [J].

Day, JW ;

Ranum, LPW .

NEUROMUSCULAR DISORDERS, 2005, 15 (01) :5-16

[9] Two-dimensional combinatorial screening identifies specific aminoglycoside - RNA internal loop partners [J].

Disney, Matthew D. ;

Labuda, Lucas P. ;

Paul, Dustin J. ;

Poplawski, Shane G. ;

Pushechnikov, Alexei ;

Tran, Tuan ;

Velagapudi, Sai P. ;

Wu, Meilan ;

Childs-Disney, Jessica L. .

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 2008, 130 (33) :11185-11194

[10] Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells [J].

Elbashir, SM ;

Harborth, J ;

Lendeckel, W ;

Yalcin, A ;

Weber, K ;

Tuschl, T .

NATURE, 2001, 411 (6836) :494-498