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USH2A Exon 13
- Target
- USH2A Exon 13
- Molecular classification
- Gene Region, Pre-mRNA splice site
01
Overview
Exon 13 of the USH2A gene is a therapeutic target for exon skipping strategies to treat Usher syndrome type IIa and retinitis pigmentosa. Antisense oligonucleotides (AONs) are used to induce skipping of this exon during mRNA splicing, resulting in an internally truncated but potentially functional usherin protein. This approach aims to bypass deleterious mutations within exon 13 while preserving overall gene function.
- Other names
- USH2A exon 13Exon 13 of USH2A gene
02
Mechanism of action
Induced exclusion ("skipping") of mutant-containing exon from mature mRNA
03
Biological functions
Pre-mRNA splicing regulation
04
Disease associations
Usher syndrome type IIaRetinitis pigmentosa
05
Safety considerations
Off-target effects of AONsImmunogenicity of AONsLimited delivery to target tissues (retina, inner ear)Long-term efficacy and safety
06
Interacting drugs
Antisense oligonucleotides (AONs)
2 more in the full profile.
07
Biomarkers
USH2A mRNA levelsUsherin protein levelsVisual function testsHearing tests
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