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RNA Splicing

RNA Splicing

Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...

Alternative RNA Splicing

Alternative RNA Splicing

Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...

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Related Experiment Video

Updated: Nov 7, 2025

Using the E1A Minigene Tool to Study mRNA Splicing Changes

Using the E1A Minigene Tool to Study mRNA Splicing Changes

Published on: April 22, 2021

RNA-Targeting Splicing Modifiers: Drug Development and Screening Assays.

Zhichao Tang¹, Junxing Zhao¹, Zach J Pearson¹

¹Department of Medicinal Chemistry, University of Kansas, Lawrence, KS 66047, USA.

Molecules (Basel, Switzerland)

|April 30, 2021

Summary

RNA splicing modifiers offer a promising new therapeutic approach for diseases linked to aberrant splicing. This review covers known modifiers, screening methods, and the chemical landscape of small-molecule splicing regulators.

Keywords:

RNA-targeting alternative splicing antisense oligonucleotide high-throughput screening small molecule splicing modifier

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Last Updated: Nov 7, 2025

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Area of Science:

Molecular Biology
Pharmacology
Genetics

Background:

RNA splicing is crucial for generating mature RNA molecules.
Aberrant RNA splicing underlies various diseases, creating a need for therapeutic interventions.
RNA splicing modifiers represent a novel pharmacological strategy.

Purpose of the Study:

To review existing RNA splicing modifiers.
To discuss methods for discovering novel splicing modifiers.
To explore the chemical diversity of small-molecule splicing modifiers.

Main Methods:

Literature review of approved and experimental splicing modifiers.
Summary of high-throughput screening techniques for identifying new modifiers.
Analysis of the chemical structures and properties of small-molecule splicing modulators.

Main Results:

Several antisense oligonucleotide splicing modifiers are FDA-approved for conditions like SMA and DMD.
Small molecules, such as risdiplam, are emerging as effective splicing regulators.
Novel RNA-targeting modifiers can address previously undruggable genetic targets.

Conclusions:

RNA splicing modulation is a viable therapeutic modality for genetic diseases and potentially infectious diseases.
Small molecules are key components in the development of RNA splicing-targeting drugs.
Further development of splicing modifiers will expand therapeutic options and research tools.