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Cat. No.: HY-147412

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Ultevursen

(Synonyms: QR-421a)

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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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Pureté: 94.24%
CAS No.: 2642382-41-8
Formule: C₂₇₃H₃₉₁N₈₁O₁₄₄P₂₀S₂₀
Masse moléculaire:8372.24

Stockage:

-20°C, sealed storage, away from moisture

* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Ultevursen Chemical Structure

CAS No.: 2642382-41-8

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Use of MCE Products

Fluorescent Dye

Strong Fluorescence Signal / Multi-Disciplinary Applications / High Fluorescence Specificity

Activité biologique
- Description
- IC₅₀ & Target
- In Vitro
- In Vivo
- Essai clinique
Chemical Information
Pureté et documentation
Références
Calculators
Help & FAQs
Classifications
Bioz

Activité biologique

Description

IC₅₀ & Target

Usherin^[1]

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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 (QR-421a) related PMO restored visual function by inducing exon skipping in ush2a-exon13 mutant zebrafish^[2].
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.

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]
Dosage:

1 μL viral suspension
Administration:

Intratympanic + utricle microinjection; single administration; harvested after 16 weeks of in vivo incubation
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.

Essai clinique

NCT Number	Sponsor	Condition	Start Date	Phase A549
NCT01329991	Plexxikon\|		2011-05	PHASE1

Chemical Information

CAS No. 2642382-41-8
Appearance Solid
Masse moléculaire 8372.24
Formule C₂₇₃H₃₉₁N₈₁O₁₄₄P₂₀S₂₀
Color White to off-white
SMILES

[Ultevursen]
Synonyms

QR-421a
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)
Livraison

Room temperature in continental US; may vary elsewhere.
Stockage

-20°C, sealed storage, away from moisture

* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Pureté et documentation

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Pureté: 99.98%

Assay: 99.98%

ee.: 99.98%

Fiche technique (271 KB)
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)
COA (2659 KB)
Elemental Analysis Report (2659 KB)
HNMR (2659 KB)
CNMR (2659 KB)
RP-HPLC (2659 KB)
NP-HPLC (2659 KB)
MS (2659 KB)
LCMS (2659 KB)
IR (2659 KB)
GC (2659 KB)
OR (2659 KB)
SDS-PAGE (2659 KB)
SEC-HPLC (2659 KB)
Other Report (2659 KB)
Instruction de manipulation (2242 KB)

Références

[1]. Girach A, et al. RNA-based therapies in inherited retinal diseases. Ther Adv Ophthalmol. 2022;14:25158414221134602. Published 2022 Vol 4:1-20. [Content Brief]

[2]. Mauriac SA, et al. AAV-mediated exon skipping therapy for Usher syndrome, type 2A. Mol Ther. 2026;34(1):179-202. [Content Brief]

Calculators

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Help & FAQs

How should lyophilized recombinant proteins be reconstituted and stored?

1. Before opening the cap, centrifuge the vial at 13000 rpm for 20-30 seconds. This step will ensure that any lyophilized powder that may have adhered to the cap or walls is collected at the bottom of the vial, minimizing the risk of product loss.

Keywords

100 mg

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Activité biologique
- Description
- IC₅₀ & Target
- In Vitro
- In Vivo
- Essai clinique
Chemical Information
Pureté et documentation
Références

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Ultevursen

Voir tous les produits spécifiques à Isoform DNA/RNA Synthesis

Activité biologique

Chemical Information

Pureté et documentation

Références

Calculators

Help & FAQs

Ultevursen Related Classifications

HY-147412 Related Classifications