Small molecules modulating RNA splicing: a review of targets and future perspectives

Bouton, Léa; Ecoutin, Agathe; Malard, Florian; Campagne, Sébastien

doi:10.1039/d3md00685a

RSC Med. Chem.

2024

DOI: 10.1039/d3md00685a

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Small molecules modulating RNA splicing: a review of targets and future perspectives

Léa Bouton,

Agathe Ecoutin,

Florian Malard

et al.

Abstract: In eukaryotic cells, RNA splicing is crucial for gene expression. Dysregulation of this process can result in incorrect mRNA processing, leading to aberrant gene expression patterns. Such abnormalities are implicated...

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Cited by 2 publications

References 156 publications

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From non-coding to coding: The importance of long non-coding RNA translation in de novo gene birth

Shiraishi,

Matsumoto

2024

Biochimica et Biophysica Acta (BBA) - General Subjects

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From non-coding to coding: The importance of long non-coding RNA translation in de novo gene birth

Shiraishi,

Matsumoto

2024

Biochimica et Biophysica Acta (BBA) - General Subjects

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Technologies for Targeted RNA Degradation and Induced RNA Decay

Mikutis,

Bernardes

2024

Chem. Rev.

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The vast majority of the human genome codes for RNA, but RNA-targeting therapeutics account for a small fraction of approved drugs. As such, there is great incentive to improve old and develop new approaches to RNA targeting. For many RNA targeting modalities, just binding is not sufficient to exert a therapeutic effect; thus, targeted RNA degradation and induced decay emerged as powerful approaches with a pronounced biological effect. This review covers the origins and advanced use cases of targeted RNA degrader technologies grouped by the nature of the targeting modality as well as by the mode of degradation. It covers both well-established methods and clinically successful platforms such as RNA interference, as well as emerging approaches such as recruitment of RNA quality control machinery, CRISPR, and direct targeted RNA degradation. We also share our thoughts on the biggest hurdles in this field, as well as possible ways to overcome them.

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Small molecules modulating RNA splicing: a review of targets and future perspectives

Abstract: In eukaryotic cells, RNA splicing is crucial for gene expression. Dysregulation of this process can result in incorrect mRNA processing, leading to aberrant gene expression patterns. Such abnormalities are implicated...

Cited by 2 publications

References 156 publications

From non-coding to coding: The importance of long non-coding RNA translation in de novo gene birth

From non-coding to coding: The importance of long non-coding RNA translation in de novo gene birth

Technologies for Targeted RNA Degradation and Induced RNA Decay

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