Ultevursen
Based on 1 Customer Validation
Ultevursen (QR-421a) is a splice-modulating antisense oligonucleotide targeting exon 13 of the USH2A gene, which restores the functional expression of Usherin protein by inducing exon skipping. Ultevursen binds to USH2A pre-mRNA and modulates the splicing process to specifically skip exon 13 carrying the pathogenic mutation c.2299delG, generating an in-frame transcript and a truncated yet functionally normal protein. Ultevursen exhibits concentration-dependent exon skipping activity in human cells and retinal organoid models, and restores Usherin expression and retinal function in zebrafish and gene-edited mouse models. Ultevursen can be used for related research on type 2 Usher syndrome and non-syndromic retinitis pigmentosa.
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- Pureza: 94.24%
- No. CAS: 2642382-41-8
- Fòrmula: C273H391N81O144P20S20
- Peso molecular:8372.24
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Almacenamiento:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Actividad biológica
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Chemical Information
- Pureza y Documentación
- Referencias
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Help & FAQs
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Actividad biológica
Descripciòn
IC50 & Target
Usherin[1]
In Vitro
Ultevursen induces concentration-dependent USH2A exon 13 skipping in WERI-Rb1 cells[1].
Ultevursen induces concentration-dependent USH2A exon 13 skipping in patient-derived iPSC-derived photoreceptor progenitor cells[1].
Ultevursen (QR-421a) (50 ng; 3 days) delivered as a U7 snRNA skipper induces USH2A exon 13 skipping in stable HEK293T USH2A-eGFP reporter cells, leading to detectable eGFP expression[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
In Vivo
Ultevursen associated AAV-U7 dual skipper (1 μL viral vector suspension; intratympanic + utricle microinjection; P1-P2 neonatal Ush2a KO-1/KO-1 mice; 16 weeks in vivo incubation period) achieves robust hair cell transduction and mediates sustained Ush2a exon skipping within mouse inner ear tissue[2].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Neonatal Ush2a KO-1/KO-2 WT/C57BL6 (P1-P2, both male and female) received intratympanic combined utricle microinjection of Ultevursen (QR-421a) associated AAV-U7 dual skipper (1 μL viral suspension per mouse)[2]
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Dosage:1 μL viral suspension
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Administration:Intratympanic + utricle microinjection; single administration; harvested after 16 weeks of in vivo incubation
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Result:Achieved robust transduction of cochlear hair cells in mouse inner ear tissues.
Immunofluorescence staining showing stable Ush2a exon skipping effect and recovered usherin protein expression within inner ear hair cells.
Ensayo clínico
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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No. CAS 2642382-41-8
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Appearance Solid
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Peso molecular 8372.24
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Fòrmula C273H391N81O144P20S20
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Color White to off-white
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SMILES
[Ultevursen]
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Synonyms
QR-421a
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Sequence
RNA, (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5C-m5U-m5U-m5C-G-G-A-G-A-A-A-m5U-m5U-m5U-A-A-A-m5U-m5C)
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Pureza y Documentación
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Ficha de datos (271 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Instrucciones de manejo (2242 KB)
Referencias
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Ultevursen Related Classifications
HY-147412 Related Classifications
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Keywords
- Ultevursen
- 2642382-41-8
- QR-421a
- Nucleoside Antimetabolite/Analog
- DNA/RNA Synthesis
- iPSC-derived photoreceptor progenitor cells
- usherin protein
- usher syndrome type 2
- usher syndrome type 2a
- HEK293T USH2A-eGFP reporter cells
- USH2A exon 13
- USH2A c.2299delG
- WERI-Rb1 cells
- USH2A pre-mRNA
- non-syndromic retinitis pigmentosa
- Inhibitor
- inhibitor
- inhibit