| Field | Specification |
|---|---|
| CAS no. | |
| Applications | |
| Molecular weight | |
| Molecular formula | C234H340N61O128P17S17 |
| Purity | |
| SMILES | |
| Form | Solid |
| Storage | |
| Shipping | |
| Catalog no. (Mfr.) | |
| Main SKU |
Compound Overview
Nusinersen is an antisense oligonucleotide active molecule that modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting production of full-length SMN protein. It improves spinal muscular atrophy[1][2][3]. It is supplied as a white to off-white solid (C234H340N61O128P17S17, MW 7127.19) at 97.56% purity.
Physical & Chemical Properties
| CAS Number | 1258984-36-9 |
|---|---|
| Molecular Formula | C234H340N61O128P17S17 |
| Molecular Weight | 7127.19 g/mol |
| Purity | 97.56% |
| Appearance | Solid |
| Color | White to off-white |
| 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) |
| 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 |
| Signaling Pathway | Cell Cycle/DNA Damage |
| Storage | -20°C, sealed storage, away from moisture. In solvent: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). |
| Shipping | Room temperature in continental US; may vary elsewhere. |
Literature Cited
Sources cited in this description and in the In Vitro & In Vivo Data tab. Peer-reviewed publications that used this product are listed under References.
Safety
For Research Use Only. Not for use in diagnostic or therapeutic procedures, and not for human or veterinary use. Handle in accordance with the Safety Data Sheet and your institution's chemical hygiene plan.
In Vivo
In a SMA mouse model, Nusinersen (6.5 mg/kg; ICV; at postnatal day 1) improves motor function and stops motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution[3].
| Animal Model | SMNΔ7 mice (Smn-/-;SMN2+/+; SMN∆7+/+, male/female), a severe SMA mouse model[3] |
|---|---|
| Dosage | 6.5 mg/kg |
| Administration | Intracerebroventricular (ICV) injection; at postnatal day 1 |
| 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. |
Data provided by the manufacturer. Numbered citations refer to the Literature Cited list in the product description.
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A sensitive analytical strategy of oligonucleotide functionalized fluorescent probes for detection of nusinersen sodium in human serum. Talanta 2024 Aug 1:275:126153. PMID: 38692053
Persistent deficits in the motor unit following mono and dual administration of SMN up-regulators in the SmnΔ7 mouse model of spinal muscular atrophy. Exp Neurol 2026 Jun 26:404:115898. PMID: 42362038
LC-MS/MS quantification of nusinersen in rat cerebrospinal fluid and preclinical pharmacokinetics study application. Bioanalysis 2025 Jul;17(13):839-846. PMID: 40689874
University of Strathclyde. 2026.
bioRxiv. 2026 Mar 30.
A therapeutic antisense oligonucleotide encompassing 2'- O-methoxyethyl modification triggers unique perturbation of the transcriptome. NAR Mol Med 2026 Jan 6;3(1):ugag002. PMID: 41551726
KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS. bioRxiv 2025 Jul 13:2025.07.11.664426. PMID: 40672150
