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RNA Splicing Modulator

Molecular classification: Antisense Oligonucleotide, Small Molecule

Overview

An RNA splicing modulator is a molecule that alters the process of pre-mRNA splicing, influencing gene expression and protein diversity. They can enhance or inhibit specific splicing events. Types include antisense oligonucleotides and small molecules. Therapeutic applications include treating genetic diseases and cancer. Molecular targets include spliceosome components, regulatory proteins, and pre-mRNA sequences.

Mechanism of action

Alters pre-mRNA processing by modifying intron removal/exon joining

Biological functions

Regulation of gene expressionPre-mRNA processingAlternative splicing modulation

Disease associations

Genetic DiseasesCancer

Interacting drugs

Risdiplam

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