Nusinersen
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[1][2][3].
Product Specifications
CAS Number
1258984-36-9
UNSPSC
12352208
Hazard Statement
H300+H310+H330
Target
DNA/RNA Synthesis
Type
Oligonucleotides
Related Pathways
Cell Cycle/DNA Damage
Applications
COVID-19-anti-virus
Field of Research
Neurological Disease
Assay Protocol
https://www.medchemexpress.com/nusinersen.html
Purity
99.62
Solubility
10 mM in H2O
Smiles
[Nusinersen]
Molecular Formula
C234H340N61O128P17S17
Molecular Weight
7127.30
Precautions
H300+H310+H330
References & Citations
[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.|[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.|[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.
Shipping Conditions
Blue Ice
Storage Conditions
-20°C (Powder, sealed storage, away from moisture)
Scientific Category
Oligonucleotides
Clinical Information
Launched
Citation 01
BioRxiv. 2025 Jul 13:2025.07.11.664426.|Bioanalysis. 2025 Jul;17 (13) :839-846.|Talanta. 2024 Aug 1:275:126153.
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