Splice-switching small molecules: a new therapeutic approach to modulate gene expression

Research output: Contribution to journalBook/Film/Article review

1 Citation (Scopus)

Abstract

Manipulating alternative RNA splicing events with small molecules is emerging as a viable mechanism for the development of therapeutics. A salient challenge in the field is understanding the molecular determinants defining the selectivity of splice-switching events and their mechanisms of action. In this review, the current state-of-the-art in splice-switching small molecules is described. Three examples of splice-switching small molecules are presented, and the differences in their modes of action compared.
LanguageEnglish
JournalMethods
Early online date14 Jun 2019
DOIs
Publication statusE-pub ahead of print - 14 Jun 2019

Fingerprint

Alternative Splicing
Gene expression
Gene Expression
Molecules
Therapeutics

Keywords

  • RNA splicing
  • small molecules
  • splice switching

Cite this

@article{990d268d4ac544aebf2b7f60fecb06e7,
title = "Splice-switching small molecules: a new therapeutic approach to modulate gene expression",
abstract = "Manipulating alternative RNA splicing events with small molecules is emerging as a viable mechanism for the development of therapeutics. A salient challenge in the field is understanding the molecular determinants defining the selectivity of splice-switching events and their mechanisms of action. In this review, the current state-of-the-art in splice-switching small molecules is described. Three examples of splice-switching small molecules are presented, and the differences in their modes of action compared.",
keywords = "RNA splicing, small molecules, splice switching",
author = "Andrea Taladriz-Sender and Emma Campbell and Burley, {Glenn A.}",
year = "2019",
month = "6",
day = "14",
doi = "10.1016/j.ymeth.2019.06.011",
language = "English",
journal = "Methods",
issn = "1046-2023",

}

TY - JOUR

T1 - Splice-switching small molecules

T2 - Methods

AU - Taladriz-Sender, Andrea

AU - Campbell, Emma

AU - Burley, Glenn A.

PY - 2019/6/14

Y1 - 2019/6/14

N2 - Manipulating alternative RNA splicing events with small molecules is emerging as a viable mechanism for the development of therapeutics. A salient challenge in the field is understanding the molecular determinants defining the selectivity of splice-switching events and their mechanisms of action. In this review, the current state-of-the-art in splice-switching small molecules is described. Three examples of splice-switching small molecules are presented, and the differences in their modes of action compared.

AB - Manipulating alternative RNA splicing events with small molecules is emerging as a viable mechanism for the development of therapeutics. A salient challenge in the field is understanding the molecular determinants defining the selectivity of splice-switching events and their mechanisms of action. In this review, the current state-of-the-art in splice-switching small molecules is described. Three examples of splice-switching small molecules are presented, and the differences in their modes of action compared.

KW - RNA splicing

KW - small molecules

KW - splice switching

UR - https://www.sciencedirect.com/journal/methods

U2 - 10.1016/j.ymeth.2019.06.011

DO - 10.1016/j.ymeth.2019.06.011

M3 - Book/Film/Article review

JO - Methods

JF - Methods

SN - 1046-2023

ER -