Target intelligence / Profile preview

RNA Splicing Machinery (Spliceosome)

Target
Spliceosome
Molecular classification
Ribonucleoprotein complex, snRNP, RNA-binding protein
01

Overview

The RNA splicing machinery, or spliceosome, is a large ribonucleoprotein complex responsible for removing introns from pre-mRNA and ligating exons. It consists of five snRNPs (U1, U2, U4, U5, U6) and over 300 associated proteins. It plays a crucial role in gene expression and proteomic diversity through alternative splicing. Dysregulation of splicing is implicated in various diseases, including cancer.

Other names
Spliceosome complexmRNA splicing machineryNuclear spliceosome
02

Mechanism of action

Modulation of RNA splicing

03

Biological functions

RNA splicingGene expression regulationmRNA processingAlternative splicing
04

Disease associations

CancerGenetic disordersNeurodegenerative diseases
05

Safety considerations

Off-target effects on splicingPotential for unintended isoform switchingComplexity of the splicing code
06

Biomarkers

Aberrant splice variantsExpression levels of splicing factors

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