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Target intelligence / Profile preview
USH2A pre-mRNA
- Target
- USH2A pre-mRNA
- Molecular classification
- RNA, pre-mRNA
01
Overview
USH2A pre-mRNA is the precursor messenger RNA transcribed from the USH2A gene, which encodes usherin, a large protein critical for normal function in the retina and inner ear. Mutations affecting USH2A pre-mRNA splicing are a major cause of Usher syndrome type II and autosomal recessive retinitis pigmentosa. It is a therapeutic target for antisense oligonucleotides to correct splicing defects.
02
Mechanism of action
Splice modulation; exon skipping; correction of aberrant transcripts
03
Biological functions
Precursor to mRNA processingGene expressionProduction of usherin protein
04
Disease associations
Usher syndrome type IIARetinitis pigmentosa
05
Safety considerations
Off-target effects of AONsSpecificity of splice modulation
06
Interacting drugs
Antisense oligonucleotides (AONs)
1 more in the full profile.
07
Biomarkers
USH2A mutationsAberrant splice variantsPseudoexon inclusion
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