Nusinersen sodium
Based on 5 publication(s) in Google Scholar
Nusinersen sodium is an antisense oligonucleotide active molecule. Nusinersen sodium modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen sodium improves spinal muscular atrophy.
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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: 98.90%
- 分子式: C234H322N61Na18O128P17S17
- 分子量:7127.3 (free acid)
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保管条件:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
MedChemExpress(MCE)の使用を引用している文献 Nusinersen sodium
MoreDNA/RNA Synthesis アイソフォーム固有の製品をすべて表示
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生物活性
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:SMNΔ7 mice (Smn-/-;SMN2+/+; SMN∆7+/+, male/female), a severe SMA mouse model[3]
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Dosage:6.5 mg/kg
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Administration:Intracerebroventricular (ICV) injection; at postnatal day 1
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Result:Improved righting reflex latency, preserved αMN number, reduced motor neuron degeneration, and rescued skeletal muscle fiber size and oxidative metabolism.
Increased SMN protein in spinal cord but not in muscle.
化学情報
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性状 Solid
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分子量 7127.3 (free acid)
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分子式 C234H322N61Na18O128P17S17
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Color White to off-white
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SMILES
[Nusinersen (sodium)]
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輸送条件
Room temperature in continental US; may vary elsewhere.
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保管条件
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Publications (5)
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Journal Impact Factor
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Most Recent
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Talanta
A sensitive analytical strategy of oligonucleotide functionalized fluorescent probes for detection of nusinersen sodium in human serum. [Abstract]2024 Aug 1:275:126153. PMID: 38692053 -
Exp Neurol
Persistent deficits in the motor unit following mono and dual administration of SMN up-regulators in the SmnΔ7 mouse model of spinal muscular atrophy. [Abstract]2026 Jun 26:404:115898. PMID: 42362038 -
Bioanalysis
LC-MS/MS quantification of nusinersen in rat cerebrospinal fluid and preclinical pharmacokinetics study application. [Abstract]2025 Jul;17(13):839-846. PMID: 40689874 -
NAR Mol Med
A therapeutic antisense oligonucleotide encompassing 2'- O-methoxyethyl modification triggers unique perturbation of the transcriptome. [Abstract]2026 Jan 6;3(1):ugag002. PMID: 41551726 -
bioRxiv
KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS. [Abstract]2025 Jul 13:2025.07.11.664426. PMID: 40672150
溶剤 & 溶解度
H2O : ≥ 10 mg/mL
* "≥" means soluble, but saturation unknown.
純度とドキュメンテーション
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データシート (270 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)
- Portuguese - PT (252 KB)
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取扱説明書 (2242 KB)
参考文献
[1]. Richard S Finkel, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732. [Content Brief]
[2]. Laura Torres-Benito, et al. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Am J Hum Genet. 2019 Jul 3;105(1):221-230. [Content Brief]
[3]. Berciano MT, et al. Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model. Sci Rep. 2020 Jul 1;10(1):10738. [Content Brief]
Calculators
濃度 (開始) × 体積 (開始) = 濃度 (終了) × 体積 (終了)