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Alternative Splicing Regulatory Machinery
- Molecular classification
- Spliceosome, RNA-binding proteins, Serine/arginine-rich proteins (SR proteins), Heterogeneous nuclear ribonucleoproteins (hnRNPs), Noncoding RNAs (e.g., lncRNAs)
01
Overview
The alternative splicing regulatory machinery encompasses the molecular components and mechanisms that govern alternative splicing, a process by which different combinations of exons are joined or skipped during pre-mRNA processing to generate multiple mRNA isoforms from a single gene. Key components include the spliceosome (snRNPs), cis-acting elements (ESE/ISE/ESS/ISS), trans-acting factors (SR proteins, hnRNPs, other RBPs), and noncoding RNAs. Dysregulation of this machinery is implicated in various diseases, making it a potential therapeutic target. Splice-switching oligonucleotides can be designed to modulate specific splicing events for therapeutic benefit.
02
Mechanism of action
Modulation of splice site selection via antisense oligonucleotides; alteration of SR protein activity by noncoding RNAs; modulation of enhancer/silencer binding
03
Biological functions
Regulation of gene expressionAlternative splicingmRNA processingProtein isoform generationTissue-specific gene expressionDevelopmental regulation
04
Disease associations
CancerGenetic disordersNeurological disordersOther
05
Safety considerations
Off-target effects of splice-switching oligonucleotidesPotential for unintended consequences of altering splicing patterns
06
Interacting drugs
Antisense oligonucleotides (splice-switching oligonucleotides)
07
Biomarkers
Aberrant splice variants as biomarkers for disease diagnosis and prognosis
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