Nusinersen
Based on 5 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.
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- Pureza: 97.56%
- No. CAS: 1258984-36-9
- Fòrmula: C234H340N61O128P17S17
- Peso molecular:7127.19
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Almacenamiento:
-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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Actividad biológica
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Chemical Information
- Pureza y Documentación
- Referencias
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Help & FAQs
Publications Citing Use of MedChemExpress (MCE) Nusinersen
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Actividad biológica
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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
|
|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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No. CAS 1258984-36-9
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Appearance Solid
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Peso molecular 7127.19
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Fòrmula 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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Sequence
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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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, 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
Pureza y Documentación
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Ficha de datos (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)
- Portuguese - PT (480 KB)
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Instrucciones de manejo (2242 KB)
Referencias
[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
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
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