2. Cell Cycle/DNA Damage
  3. Nucleoside Antimetabolite/Analog DNA/RNA Synthesis
  4. Ultevursen

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.

For research use only. We do not sell to patients.

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)

Ultevursen Chemical Structure

CAS No. : 2642382-41-8

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Based on 1 publication(s) in Google Scholar

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Description

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[1][2].

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.

Ultevursen Related Antibodies
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.
Clinical Trial
Molecular Weight

8372.24

Formula

C273H391N81O144P20S20
CAS No.
Appearance

Solid

Color

White to off-white

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)
SMILES

[Ultevursen]
Shipping

Room temperature in continental US; may vary elsewhere.
Storage

-20°C, sealed storage, away from moisture

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

Purity & Documentation
References
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  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

Keywords:

Ultevursen2642382-41-8QR-421aNucleoside Antimetabolite/AnalogDNA/RNA SynthesisiPSC-derived photoreceptor progenitor cellsusherin proteinusher syndrome type 2usher syndrome type 2aHEK293T USH2A-eGFP reporter cellsUSH2A exon 13USH2A c.2299delGWERI-Rb1 cellsUSH2A pre-mRNAnon-syndromic retinitis pigmentosaInhibitorinhibitorinhibit

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