For research use only. Not for therapeutic Use.
Nusinersen（Cat No.:I033709）is an antisense oligonucleotide therapy used to treat spinal muscular atrophy (SMA), a genetic disorder characterized by progressive muscle weakness and atrophy. It works by increasing the production of functional survival motor neuron (SMN) protein, which is deficient in SMA patients due to mutations in the SMN1 gene. Nusinersen targets the SMN2 gene, promoting the inclusion of exon 7 in its mRNA, thereby boosting SMN protein levels. Administered via intrathecal injection, nusinersen has shown significant improvements in motor function and survival, offering a critical treatment option for SMA patients.
| Catalog Number | I033709 |
| CAS Number | 1258984-36-9 |
| Synonyms | ASO-10-27; ISIS 396443; ISIS SMNRx; Nusinersen |
| Molecular Formula | C234H340N61O128P17S17 |
| Purity | 98% |
| Target | DNA/RNA Synthesis |
| Target Protein | |
| Solubility | Soluble in DMSO |
| Appearance | Solid |
| Storage | Dry, dark and at 2 - 8 °C for six months or -20°C for two years. |
| IUPAC Name | 1-[(2R,3R,4R,5R)-4-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[(2R,3R,4R,5R)-5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(2-amino-6-oxo-1H-purin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(6-aminopurin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(6-aminopurin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(6-aminopurin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(6-aminopurin-9-yl)-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-4-(2-methoxyethoxy)oxolan-2-yl]methoxy-hydroxyphosphinothioyl]oxy-5-(hydroxymethyl)-3-(2-methoxyethoxy)oxolan-2-yl]-5-methylpyrimidine-2,4-dione |
| InChI | InChI=1S/C234H340N61O128P17S17/c1-106-66-278(224(308)261-178(106)235)205-162(356-50-32-338-14)144(119(391-205)79-373-425(320,442)407-143-117(77-296)389-204(161(143)355-49-31-337-13)282-70-110(5)193(298)271-228(282)312)410-431(326,448)383-89-129-154(172(366-60-42-348-24)215(401-129)289-99-254-135-182(239)246-95-250-186(135)289)419-436(331,453)379-81-121-145(163(357-51-33-339-15)206(393-121)279-67-107(2)179(236)262-225(279)309)408-426(321,443)375-83-123-149(167(361-55-37-343-19)210(395-123)284-72-112(7)195(300)273-230(284)314)413-429(324,446)378-86-125-150(168(362-56-38-344-20)211(397-125)285-73-113(8)196(301)274-231(285)315)414-430(325,447)377-85-124-148(166(360-54-36-342-18)209(396-124)283-71-111(6)194(299)272-229(283)313)412-428(323,445)374-80-120-147(165(359-53-35-341-17)208(392-120)281-69-109(4)181(238)264-227(281)311)411-432(327,449)384-90-130-155(173(367-61-43-349-25)216(402-130)290-100-255-136-183(240)247-96-251-187(136)290)420-437(332,454)381-87-127-152(170(364-58-40-346-22)213(399-127)287-75-115(10)198(303)276-233(287)317)415-433(328,450)385-91-131-157(175(369-63-45-351-27)218(403-131)292-102-257-138-185(242)249-98-253-189(138)292)422-440(335,457)386-92-132-156(174(368-62-44-350-26)217(404-132)291-101-256-137-184(241)248-97-252-188(137)291)421-438(333,455)382-88-128-153(171(365-59-41-347-23)214(400-128)288-76-116(11)199(304)277-234(288)318)417-435(330,452)388-94-134-159(177(371-65-47-353-29)220(406-134)295-105-260-141-192(295)267-223(245)270-202(141)307)423-439(334,456)380-82-122-146(164(358-52-34-340-16)207(394-122)280-68-108(3)180(237)263-226(280)310)409-427(322,444)376-84-126-151(169(363-57-39-345-21)212(398-126)286-74-114(9)197(302)275-232(286)316)416-434(329,451)387-93-133-158(176(370-64-46-352-28)219(405-133)294-104-259-140-191(294)266-222(244)269-201(140)306)418-424(319,441)372-78-118-142(297)160(354-48-30-336-12)203(390-118)293-103-258-139-190(293)265-221(243)268-200(139)305/h66-76,95-105,117-134,142-177,203-220,296-297H,30-65,77-94H2,1-29H3,(H,319,441)(H,320,442)(H,321,443)(H,322,444)(H,323,445)(H,324,446)(H,325,447)(H,326,448)(H,327,449)(H,328,450)(H,329,451)(H,330,452)(H,331,453)(H,332,454)(H,333,455)(H,334,456)(H,335,457)(H2,235,261,308)(H2,236,262,309)(H2,237,263,310)(H2,238,264,311)(H2,239,246,250)(H2,240,247,251)(H2,241,248,252)(H2,242,249,253)(H,271,298,312)(H,272,299,313)(H,273,300,314)(H,274,301,315)(H,275,302,316)(H,276,303,317)(H,277,304,318)(H3,243,265,268,305)(H3,244,266,269,306)(H3,245,267,270,307)/t117-,118-,119-,120-,121-,122-,123-,124-,125-,126-,127-,128-,129-,130-,131-,132-,133-,134-,142-,143-,144-,145-,146-,147-,148-,149-,150-,151-,152-,153-,154-,155-,156-,157-,158-,159-,160-,161-,162-,163-,164-,165-,166-,167-,168-,169-,170-,171-,172-,173-,174-,175-,176-,177-,203-,204-,205-,206-,207-,208-,209-,210-,211-,212-,213-,214-,215-,216-,217-,218-,219-,220-,424?,425?,426?,427?,428?,429?,430?,431?,432?,433?,434?,435?,436?,437?,438?,439?,440?/m1/s1 |
| InChIKey | WWFDJIVIDXJAQR-FFWSQMGZSA-N |
| SMILES | CC1=CN(C(=O)NC1=O)C2C(C(C(O2)CO)OP(=S)(O)OCC3C(C(C(O3)N4C=C(C(=NC4=O)N)C)OCCOC)OP(=S)(O)OCC5C(C(C(O5)N6C=NC7=C(N=CN=C76)N)OCCOC)OP(=S)(O)OCC8C(C(C(O8)N9C=C(C(=NC9=O)N)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=NC1=O)N)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C(N=CN=C21)N)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C(N=CN=C21)N)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C(N=CN=C21)N)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C1N=C(NC2=O)N)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=NC1=O)N)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=C(C(=O)NC1=O)C)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C1N=C(NC2=O)N)OCCOC)OP(=S)(O)OCC1C(C(C(O1)N1C=NC2=C1N=C(NC2=O)N)OCCOC)O)OCCOC |
| Reference | 1. Chiriboga CA. Nusinersen for the treatment of spinal muscular atrophy. Expert Rev Neurother. 2017 Oct;17(10):955-962. doi: 10.1080/14737175.2017.1364159. Epub 2017 Sep 8. PMID: 28884620. |
