Cas No.: | 1258984-36-9 |
Chemical Name: | Nusinersen |
Synonyms: | Nusinersen;ISIS SMNRx;ISIS-SMN(Rx);Nusinersen [USAN:INN];5Z9SP3X666;RNA, (2'-0-(2-methoxyethyi))(p-thio)(m5u-c-a-c-m5u-m5u-m5u-c-a-m5ua- a-m5 u-g-c-m5u-g-g) |
SMILES: | S=P(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C=NC3C(N)=NC=NC2=3)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C(NC(C(C)=C2)=O)=O)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C=NC3C(NC(N)=NC2=3)=O)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C(N=C(C(C)=C2)N)=O)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C(NC(C(C)=C2)=O)=O)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C=NC3C(NC(N)=NC2=3)=O)O1)OCCOC)OP(O)(OC[C@@H]1[C@H]([C@H]([C@H](N2C=NC3C(NC(N)=NC2=3)=O)O1)OCCOC)O)=S)=S)=S)=S)=S)=S)O[C@@H]1[C@@H](COP(O)(O[C@@H]2[C@@H](COP(O)(O[C@@H]3[C@@H](COP(O)(O[C@@H]4[C@@H](COP(O)(O[C@@H]5[C@@H](COP(O)(O[C@@H]6[C@@H](COP(O)(O[C@@H]7[C@@H](COP(O)(O[C@@H]8[C@@H](COP(O)(O[C@@H]9[C@@H](COP(O)(O[C@@H]%10[C@@H](COP(O)(O[C@@H]%11[C@@H](CO)O[C@H]([C@@H]%11OCCOC)N%11C(NC(C(C)=C%11)=O)=O)=S)O[C@H]([C@@H]%10OCCOC)N%10C(N=C(C(C)=C%10)N)=O)=S)O[C@H]([C@@H]9OCCOC)N9C=NC%10C(N)=NC=NC9=%10)=S)O[C@H]([C@@H]8OCCOC)N8C(N=C(C(C)=C8)N)=O)=S)O[C@H]([C@@H]7OCCOC)N7C(NC(C(C)=C7)=O)=O)=S)O[C@H]([C@@H]6OCCOC)N6C(NC(C(C)=C6)=O)=O)=S)O[C@H]([C@@H]5OCCOC)N5C(NC(C(C)=C5)=O)=O)=S)O[C@H]([C@@H]4OCCOC)N4C(N=C(C(C)=C4)N)=O)=S)O[C@H]([C@@H]3OCCOC)N3C=NC4C(N)=NC=NC3=4)=S)O[C@H]([C@@H]2OCCOC)N2C(NC(C(C)=C2)=O)=O)=S)O[C@H]([C@@H]1OCCOC)N1C=NC2C(N)=NC=NC1=2 |
Formula: | C234H340N61O128P17S17 |
M.Wt: | 7127.1942691803 |
Purity: | >95% |
Sotrage: | 2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO |
Target: | SMN2 |
In Vitro: | Nusinersen is an SMN-ASO that increases SMN levels by blocking an intronic splice silencer in SMN2, thereby facilitating the exon 7 inclusion and generation of FL-SMN2 transcripts. |