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Nusinersen CAS NO.1258984-36-9
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Keywords
- 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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Quick Details
- ProName: Nusinersen
- CasNo: 1258984-36-9
- Molecular Formula: C234H340N61O128P17S17
- Appearance: White to Off-white Solid
- Application: the treatment of SMA
- PackAge: 5 mg
- Purity: ≥95%
- Storage: Store at -20°C
- LimitNum: 0
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Details
Nusinersen is a modified antisense oligonucleotide, where the 2'-hydroxy groups of the ribofuranosyl rings are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages are replaced with phosphorothioate linkages. Nusinersen binds to a specific sequence in the intron downstream of exon 7 of the SMN2 transcript. Nusinersen is a drug used for the treatment of spinal muscular atrophy (SMA), which is related to the mutation in the SMN1 gene. It was approved by FDA in Dec. 2016 for the treatment of SMA in adults and children.
