CAS No. 1258984-36-9
Chemical Structure
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Nusinersen
product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.
Chemical Information
| CAS Number | 1258984-36-9 | |
|---|---|---|
| Molecular Formula | ||
| Molecular Weight (MW) | 7124.42 |
Biological Activity
Nusinersen is an antisense oligonucleotide that targets the intronic splice silencer N1 site within intron 7 of SMN2 pre-mRNA. By displacement of splicing repressors hnRNP A1 and A2, it prevents splicing inhibition and promotes the inclusion of exon 7. This alternative splicing modification increases the production of full-length, functional survival motor neuron protein, rescuing motor neuron function and suppressing pathological phenotypes associated with spinal muscular atrophy. [1]
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Note: Technical data last updated: Sep 1, 2026.