Viltolarsen (NS-065/NCNP-01)   中文名称: 维特塞普索

影响年轻男孩的最普遍、最致命的肌肉萎缩性疾病之一被称为杜氏肌营养不良症 (DMD)，它是由 DMD 基因突变引起的。外显子跳跃已成为一种可行的 DMD 治疗选择。为了恢复阅读框并允许肌营养不良蛋白产生，反义寡核苷酸 (AON) 被设计为导致外显子跳跃 [1]。

[1]. Dzierlega K, et al. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther. 2020;27(9):407-416.

另见：Viltolarsen（注释已移至）。

6923.363

2055732-84-6

154562176

White to off-white solid powder

-33.1

2230

29

149

122

465

21200

42

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ZVXLKCOOOKREJD-OERAVCCFSA-N

InChI=1S/C244H381N113O88P20/c1-140-62-343(238(378)290-213(140)359)180-94-322(447(386,298(10)11)405-110-147-60-259-61-174(425-147)337-54-48-168(245)272-232(337)372)74-153(431-180)117-412-454(393,305(24)25)325-78-157(435-183(97-325)346-65-143(4)216(362)293-241(346)381)119-414-460(399,311(36)37)331-84-163(441-189(103-331)352-134-266-197-207(352)278-226(253)284-220(197)366)125-418-457(396,308(30)31)328-80-159(437-186(100-328)349-68-146(7)219(365)296-244(349)384)121-416-462(401,313(40)41)333-87-167(445-191(105-333)354-136-268-199-209(354)280-228(255)286-222(199)368)129-424-465(404,316(46)47)336-88-165(443-194(108-336)357-139-271-202-212(357)283-231(258)289-225(202)371)127-422-459(398,310(34)35)330-81-160(438-188(102-330)351-133-265-196-204(252)261-131-263-206(196)351)122-420-458(397,309(32)33)329-82-161(439-187(101-329)350-132-264-195-203(251)260-130-262-205(195)350)123-421-463(402,314(42)43)334-85-164(442-193(106-334)356-138-270-201-211(356)282-230(257)288-224(201)370)126-419-456(395,307(28)29)327-76-155(433-185(99-327)348-67-145(6)218(364)295-243(348)383)116-411-450(389,301(16)17)320-73-152(430-178(92-320)341-58-52-172(249)276-236(341)376)114-409-453(392,304(22)23)324-77-156(434-182(96-324)345-64-142(3)215(361)292-240(345)380)118-413-455(394,306(26)27)326-79-158(436-184(98-326)347-66-144(5)217(363)294-242(347)382)120-415-461(400,312(38)39)332-86-166(444-190(104-332)353-135-267-198-208(353)279-227(254)285-221(198)367)128-423-464(403,315(44)45)335-83-162(440-192(107-335)355-137-269-200-210(355)281-229(256)287-223(200)369)124-417-451(390,302(18)19)321-71-150(428-179(93-321)342-59-53-173(250)277-237(342)377)112-407-448(387,299(12)13)318-72-151(429-177(90-318)340-57-51-171(248)275-235(340)375)113-408-452(391,303(20)21)323-75-154(432-181(95-323)344-63-141(2)214(360)291-239(344)379)115-410-449(388,300(14)15)319-70-149(427-176(91-319)339-56-50-170(247)274-234(339)374)111-406-446(385,297(8)9)317-69-148(109-358)426-175(89-317)338-55-49-169(246)273-233(338)373/h48-59,62-68,130-139,147-167,174-194,259,358H,60-61,69-129H2,1-47H3,(H2,245,272,372)(H2,246,273,373)(H2,247,274,374)(H2,248,275,375)(H2,249,276,376)(H2,250,277,377)(H2,251,260,262)(H2,252,261,263)(H,290,359,378)(H,291,360,379)(H,292,361,380)(H,293,362,381)(H,294,363,382)(H,295,364,383)(H,296,365,384)(H3,253,278,284,366)(H3,254,279,285,367)(H3,255,280,286,368)(H3,256,281,287,369)(H3,257,282,288,370)(H3,258,283,289,371)/t147-,148-,149-,150-,151-,152-,153-,154-,155-,156-,157-,158-,159-,160-,161-,162-,163-,164-,165-,166-,167-,174+,175+,176+,177+,178+,179+,180+,181+,182+,183+,184+,185+,186+,187+,188+,189+,190+,191+,192+,193+,194+,446?,447?,448?,449?,450?,451?,452?,453?,454?,455?,456?,457?,458?,459?,460?,461?,462?,463?,464?,465?/m0/s1

1-[(2R,6S)-6-[[[(2S,6R)-2-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2S,6R)-2-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2S,6R)-2-[[[(2S,6R)-2-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2S,6R)-2-[[[(2S,6R)-2-[[[(2S,6R)-2-[[[(2S,6R)-2-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2R,6S)-2-(2-amino-6-oxo-1H-purin-9-yl)-6-[[[(2R,6S)-2-(4-amino-2-oxopyrimidin-1-yl)-6-[[[(2R,6S)-2-(4-amino-2-oxopyrimidin-1-yl)-6-[[[(2S,6R)-2-[[[(2R,6S)-2-(4-amino-2-oxopyrimidin-1-yl)-6-[[[(2R,6S)-2-(4-amino-2-oxopyrimidin-1-yl)-6-(hydroxymethyl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(4-amino-2-oxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(6-aminopurin-9-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(6-aminopurin-9-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-6-(5-methyl-2,4-dioxopyrimidin-1-yl)morpholin-4-yl]-(dimethylamino)phosphoryl]oxymethyl]-4-[[(2S,6R)-6-(4-amino-2-oxopyrimidin-1-yl)morpholin-2-yl]methoxy-(dimethylamino)phosphoryl]morpholin-2-yl]-5-methylpyrimidine-2,4-dione

2934.99.9001

Powder      -20°C    3 years

                     4°C     2 years

In solvent   -80°C    6 months

                  -20°C    1 month

注意： 请将本产品存放在密封且受保护的环境中（例如氮气保护），避免吸湿/受潮。

Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)

H2O : ~100 mg/mL (~14.44 mM)

配方 1 中的溶解度: 50 mg/mL (7.22 mM) in PBS (这些助溶剂从左到右依次添加，逐一添加), 澄清溶液; 超声助溶。

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请根据您的实验动物和给药方式选择适当的溶解配方/方案：
1、请先配制澄清的储备液(如：用DMSO配置50 或 100 mg/mL母液(储备液));
2、取适量母液，按从左到右的顺序依次添加助溶剂，澄清后再加入下一助溶剂。以 下列配方为例说明 (注意此配方只用于说明，并不一定代表此产品 的实际溶解配方):
10% DMSO → 40% PEG300 → 5% Tween-80 → 45% ddH2O (或 saline);
假设最终工作液的体积为 1 mL, 浓度为5 mg/mL: 取 100 μL 50 mg/mL 的澄清 DMSO 储备液加到 400 μL PEG300 中，混合均匀/澄清；向上述体系中加入50 μL Tween-80，混合均匀/澄清；然后继续加入450 μL ddH2O (或 saline)定容至 1 mL；
3、溶剂前显示的百分比是指该溶剂在最终溶液/工作液中的体积所占比例;
4、 如产品在配制过程中出现沉淀/析出，可通过加热(≤50℃)或超声的方式助溶;
5、为保证最佳实验结果，工作液请现配现用！
6、如不确定怎么将母液配置成体内动物实验的工作液，请查看说明书或联系我们;
7、 以上所有助溶剂都可在 Invivochem.cn网站购买。