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

Andrea Taladriz-Sender; Emma Campbell; Glenn A. Burley

doi:10.1016/j.ymeth.2019.06.011

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

Andrea Taladriz-Sender, Emma Campbell, Glenn A. Burley

Pure And Applied Chemistry

Research output: Contribution to journal › Book/Film/Article review › peer-review

8 Citations (Scopus)

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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.

Original language	English
Pages (from-to)	134-142
Number of pages	9
Journal	Methods
Volume	167
Early online date	14 Jun 2019
DOIs	https://doi.org/10.1016/j.ymeth.2019.06.011
Publication status	Published - 1 Sept 2019

Keywords

RNA splicing
small molecules
splice switching

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10.1016/j.ymeth.2019.06.011

Taladriz-Sender-etal-Methods-2019-Splice-switching-small-molecules-a-new-therapeutic-approachAccepted author manuscript, 1.24 MBLicence: CC BY-NC-ND 4.0

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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. ",

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AU - Burley, Glenn A.

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Y1 - 2019/9/1

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.

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KW - small molecules

KW - splice switching

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U2 - 10.1016/j.ymeth.2019.06.011

DO - 10.1016/j.ymeth.2019.06.011

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EP - 142

JO - Methods

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ER -

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

Abstract

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Regulation of alternative splicing by G-quadruplexes: molecular mechanisms and tools to manipulate gene expression

Data for: "A Chemo- and Regioselective Tandem [3+2]Heteroannulation Strategy for Carbazole Synthesis: Synergizing Two Mechanistically Distinct Bond-Forming Processes"

A chemo- and regioselective tandem [3+2]heteroannulation strategy for carbazole synthesis: combining two mechanistically distinct bond-forming processes

Cite this

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

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Projects

Regulation of alternative splicing by G-quadruplexes: molecular mechanisms and tools to manipulate gene expression

Datasets

Data for: "A Chemo- and Regioselective Tandem [3+2]Heteroannulation Strategy for Carbazole Synthesis: Synergizing Two Mechanistically Distinct Bond-Forming Processes"

Research output

A chemo- and regioselective tandem [3+2]heteroannulation strategy for carbazole synthesis: combining two mechanistically distinct bond-forming processes

Cite this