Nusinersen
Based on 7 publication(s) in Google Scholar
Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy.
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 : 97.56%
- CAS 番号: 1258984-36-9
- 分子式: C234H340N61O128P17S17
- 分子量:7127.19
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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)
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生物活性
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化学情報
- 純度とドキュメンテーション
- 参考文献
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Help & FAQs
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MedChemExpress（MCE）の使用を引用している文献 Nusinersen
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
製品説明
体内実験
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.
臨床実験
| NCT Number | Sponsor | Condition | Start Date |
Phase
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|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
化学情報
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CAS 番号 1258984-36-9
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性状 Solid
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分子量 7127.19
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分子式 C234H340N61O128P17S17
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Color White to off-white
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SMILES
COCCO[C@@H]1[C@@H]([C@H](O[C@H]1n2c3c(C(NC(N)=N3)=O)nc2)COP(O)(O[C@@H]4[C@H](O[C@@H](n5c6c(C(NC(N)=N6)=O)nc5)[C@@H]4OCCOC)COP(S)(O[C@@H]7[C@H](O[C@@H](N8C=C(C(NC8=O)=O)C)[C@@H]7OCCOC)COP(O)(O[C@@H]9[C@H](O[C@@H](N%10C=C(C(N)=NC%10=O)C)[C@@H]9OCCOC)COP(O)(O[C@@H]%11[C@H](O[C@@H](n%12c%13c(C(NC(N)=N%13)=O)nc%12)[C@@H]%11OCCOC)COP(O)(O[C@@H]%14[C@H](O[C@@H](N%15C=C(C(NC%15=O)=O)C)[C@@H]%14OCCOC)COP(O)(O[C@@H]%16[C@H](O[C@@H](n(cn%17)c%18c%17c(N)ncn%18)[C@@H]%16OCCOC)COP(O)(O[C@@H]%19[C@H](O[C@@H](n(cn%20)c%21c%20c(N)ncn%21)[C@@H]%19OCCOC)COP(O)(O[C@@H]%22[C@H](O[C@@H](N%23C=C(C(NC%23=O)=O)C)[C@@H]%22OCCOC)COP(O)(O[C@@H]%24[C@H](O[C@@H](n(cn%25)c%26c%25c(N)ncn%26)[C@@H]%24OCCOC)COP(O)(O[C@@H]%27[C@H](O[C@@H](N%28C=C(C(N)=NC%28=O)C)[C@@H]%27OCCOC)COP(O)(O[C@@H]%29[C@H](O[C@@H](N%30C=C(C(NC%30=O)=O)C)[C@@H]%29OCCOC)COP(O)(O[C@@H]%31[C@H](O[C@@H](N%32C=C(C(NC%32=O)=O)C)[C@@H]%31OCCOC)COP(O)(O[C@@H]%33[C@H](O[C@@H](N%34C=C(C(NC%34=O)=O)C)[C@@H]%33OCCOC)COP(O)(O[C@@H]%35[C@H](O[C@@H](N%36C=C(C(N)=NC%36=O)C)[C@@H]%35OCCOC)COP(O)(O[C@@H]%37[C@H](O[C@@H](n(cn%38)c%39c%38c(N)ncn%39)[C@@H]%37OCCOC)COP(O)(O[C@@H]%40[C@H](O[C@@H](N%41C=C(C(N)=NC%41=O)C)[C@@H]%40OCCOC)COP(O)(O[C@@H]%42[C@H](O[C@@H](N%43C=C(C(NC%43=O)=O)C)[C@@H]%42OCCOC)CO)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=S)=O)=S)O
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配列
RNA, [2'-O-(2-methoxyethyl)](P-thio)(m5U-m5C-A-m5C-m5U-m5U-m5U-m5C-A-m5U-A-A-m5U-G-m5C-m5U-G-G)
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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, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Publications (7)
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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 -
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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
純度とドキュメンテーション
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データシート (271 KB)
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SDS (480 KB)
- English - EN (480 KB)
- Français - FR (480 KB)
- Deutsch - DE (480 KB)
- Norwegian - NO (480 KB)
- Español - ES (480 KB)
- Swedish - SV (480 KB)
- Italian - IT (480 KB)
- Korean - KR (480 KB)
- Portuguese - PT (480 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)
Nusinersen 関連分類
HY-112980 関連分類
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