Nusinersen sodium
Based on 6 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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- Pureza: 98.90%
- Fòrmula: C234H322N61Na18O128P17S17
- Peso molecular:7127.3 (free acid)
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Almacenamiento:
-20°C, stored under nitrogen, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
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Actividad biológica
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Chemical Information
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Solvente y solubilidad
- Pureza y Documentación
- Referencias
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Help & FAQs
Publications Citing Use of MedChemExpress (MCE) Nusinersen sodium
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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.
Chemical Information
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Appearance Solid
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Peso molecular 7127.3 (free acid)
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Fòrmula C234H322N61Na18O128P17S17
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Color White to off-white
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SMILES
[Nusinersen (sodium)]
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
-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 (6)
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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
Solvente y solubilidad
H2O : ≥ 10 mg/mL
* "≥" means soluble, but saturation unknown.
Pureza y Documentación
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Ficha de datos (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)
- Korean - KR (252 KB)
- Portuguese - PT (252 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)
Nusinersen sodium Related Classifications
HY-112980A Related Classifications
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