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製品番号: HY-147412

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Ultevursen

Name: Ultevursen
Brand: MedChemExpress
SKU: HY-147412
Price: 105.00 USD
Availability: InStock
Rating: 5 (1 reviews)

(別名: 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.

商品は「研究用試薬」です。人や動物の医療用・臨床診断用・食品用の製品ではありません。
研究用途以外に使用した場合、当社は一切の責任を負いかねます。

純度 : 94.24%
CAS 番号: 2642382-41-8
分子式: C₂₇₃H₃₉₁N₈₁O₁₄₄P₂₀S₂₀
分子量:8372.24

保管条件:

-20°C, sealed storage, away from moisture

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

Ultevursen 構造式

CAS 番号: 2642382-41-8

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Top Publications Citing

Use of MCE Products

Fluorescent Dye

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

生物活性
- 製品説明
- IC₅₀ & Target
- 体外実験
- 体内実験
- 臨床実験
化学情報
純度とドキュメンテーション
参考文献
Calculators
Help & FAQs
関連分類
Bioz

生物活性

製品説明

IC₅₀ & Target

Usherin^[1]

DNA/RNA Synthesis アイソフォーム固有の製品をすべて表示 :

体外実験

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.

体内実験

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.

Related Protocols :

臨床実験

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

化学情報

CAS 番号 2642382-41-8
性状 Solid
分子量 8372.24
分子式 C₂₇₃H₃₉₁N₈₁O₁₄₄P₂₀S₂₀
Color White to off-white
SMILES

[Ultevursen]
別名

QR-421a
配列

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)
輸送条件

Room temperature in continental US; may vary elsewhere.
保管条件

-20°C, sealed storage, away from moisture

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

純度とドキュメンテーション

バッチを選択:

純度: 99.98%

Assay: 99.98%

ee.: 99.98%

データシート (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)
元素分析報告書 (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)
取扱説明書 (2242 KB)

参考文献

[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

モル濃度計算機

Mass (g) = Concentration (mol/L) × Volume (L) × Molecular Weight (g/mol)

Mass Mass

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分子量分子量

希釈計算機

濃度 _(開始) × 体積 _(開始) = 濃度 _(終了) × 体積 _(終了)

濃度 _(開始) 濃度 _(開始)

体積 _(開始) 体積 _(開始)

濃度 _(終了) 濃度 _(終了)

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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.

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100 mg

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