A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways

Raphael I. Benhamou; Alicia J. Angelbello; Eric T. Wang; Matthew D. Disney

doi:10.1016/j.chembiol.2020.01.003

A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways

Raphael I. Benhamou, Alicia J. Angelbello, Eric T. Wang, Matthew D. Disney^*

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

15 Scopus citations

Abstract

RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.

Original language	English
Pages (from-to)	223-231.e4
Journal	Cell Chemical Biology
Volume	27
Issue number	2
DOIs	https://doi.org/10.1016/j.chembiol.2020.01.003
State	Published - 20 Feb 2020
Externally published	Yes

Bibliographical note

Publisher Copyright:
© 2020 Elsevier Ltd

Keywords

RNA
chemical biology
click chemistry
drug design
intron retention
medicinal chemistry
microsatellite disease
myotonic dystrophy
nucleic acids
repeat expansion disorder

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

Access to Document

10.1016/j.chembiol.2020.01.003

Cite this

@article{f148f28b5d44434d897ee3c80c457614,

title = "A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways",

abstract = "RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.",

keywords = "RNA, chemical biology, click chemistry, drug design, intron retention, medicinal chemistry, microsatellite disease, myotonic dystrophy, nucleic acids, repeat expansion disorder",

author = "Benhamou, {Raphael I.} and Angelbello, {Alicia J.} and Wang, {Eric T.} and Disney, {Matthew D.}",

note = "Publisher Copyright: {\textcopyright} 2020 Elsevier Ltd",

year = "2020",

month = feb,

day = "20",

doi = "10.1016/j.chembiol.2020.01.003",

language = "אנגלית",

volume = "27",

pages = "223--231.e4",

journal = "Cell Chemical Biology",

issn = "2451-9456",

publisher = "Elsevier Inc.",

number = "2",

}

TY - JOUR

T1 - A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways

AU - Benhamou, Raphael I.

AU - Angelbello, Alicia J.

AU - Wang, Eric T.

AU - Disney, Matthew D.

PY - 2020/2/20

Y1 - 2020/2/20

N2 - RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.

AB - RNA repeat expansions cause >30 diseases, and both oligonucleotides and small molecules have been developed to inhibit their dysfunction. Benhamou et al. show for the first time that small molecules targeting structured, disease-causing RNAs can shunt them toward native decay pathways by affecting their processing.

KW - RNA

KW - chemical biology

KW - click chemistry

KW - drug design

KW - intron retention

KW - medicinal chemistry

KW - microsatellite disease

KW - myotonic dystrophy

KW - nucleic acids

KW - repeat expansion disorder

UR - http://www.scopus.com/inward/record.url?scp=85078883462&partnerID=8YFLogxK

U2 - 10.1016/j.chembiol.2020.01.003

DO - 10.1016/j.chembiol.2020.01.003

M3 - ???researchoutput.researchoutputtypes.contributiontojournal.article???

C2 - 31981476

AN - SCOPUS:85078883462

SN - 2451-9456

VL - 27

SP - 223-231.e4

JO - Cell Chemical Biology

JF - Cell Chemical Biology

IS - 2

ER -

A Toxic RNA Catalyzes the Cellular Synthesis of Its Own Inhibitor, Shunting It to Endogenous Decay Pathways

Abstract

Bibliographical note

Keywords

UN SDGs

Access to Document

Other files and links

Fingerprint

Cite this