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Pre-mRNA Splicing (Exon 53 Skipping) (N/A)

Target: N/A
Molecular classification: RNA processing, Spliceosome, snRNPs

Overview

Pre-mRNA splicing is a fundamental process in eukaryotic gene expression involving the removal of introns and joining of exons. Exon skipping, particularly exon 53 skipping, is a therapeutic strategy using antisense oligonucleotides to modulate splicing and restore a partially functional protein, such as dystrophin in Duchenne muscular dystrophy. The spliceosome, composed of snRNPs, catalyzes this process, and errors can lead to various diseases.

Other names: Exon skippingAlternative splicingSpliceosome-mediated RNA splicingExon 53 skipping

Mechanism of action

Antisense oligonucleotides bind to pre-mRNA, masking splice sites and inducing exon skipping.

Biological functions

Gene expression regulationmRNA processingProtein isoform generation

Disease associations

Duchenne Muscular DystrophyGenetic disorders related to splicing defects

Safety considerations

Off-target effects of ASOsImmune response to ASOsVariable efficacy depending on patient genotypeLong-term safety of exon-skipped proteins

Interacting drugs

Antisense oligonucleotides (ASOs)

3 more in the full profile.

Biomarkers

Dystrophin protein levelsExon skipping efficiencySerum creatine kinase (CK) levelsMuscle function tests

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