Antisense Oligonucleotides
(反义寡核苷酸)
反义寡核苷酸（ASOs）通常指的是短小的、人工合成的单链DNA或RNA（13至30个核苷酸）。在与靶向的mRNA或前体mRNA结合后，ASOs通过多种不同的机制调节RNA的功能。
1. 反义寡核苷酸（ASOs）能够形成一种RNA-DNA的杂合体，该杂合体成为RNaseH的作用底物，从而导致靶mRNA的降解。
2. 反义寡核苷酸（ASOs）能够通过阻断核糖体机器的立体结构来调节基因表达，这可能导致表达量降低、剪接方式的改变以及功能性蛋白质的恢复。
3. 反义寡核苷酸（ASOs）与前体mRNA的结合能够改变剪接因子的募集情况，并调控剪接过程。
反义寡核苷酸（ASOs）的首次体内应用显示出有限的临床潜力，这是因为未修饰的磷酸核糖骨架上的ASO易受内切核酸酶和外切核酸酶的快速降解作用影响。骨架和糖分子的修饰使ASO更具亲和力和稳定性。磷酰亚胺胺糖醛酸寡聚体（PMO）对核酸酶和蛋白酶的降解具有很强的抵抗力，主要用于剪接调节或翻译抑制。在核糖环的 2' 位置进行化学修饰，如 2'-O-甲基（2'-O-me）、2'-氟（2'-F）、2'-O-甲氧基乙基（2'-MOE），使得寡核苷酸能够采用类似于RNA的 C3'-内糖构象，从而使其具有热稳定性。
Antisense Oligonucleotides (188):
| Cat. No. |
Product Name |
CAS No. |
Purity |
Chemical Structure |
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- HY-109528
-
|
Fomivirsen sodium
|
160369-77-7 |
≥99.0% |
|
Fomivirsen (ISIS-2922) 是一种反义21 mer 磷酸硫寡核苷酸。Fomivirsen sodium 是一种用于巨细胞病毒视网膜炎 (CMV) 研究的抗病毒试剂 (antiviral agent)。Fomivirsen sodium 结合并降解 CMV immediate-early 2 蛋白的 mRNA，从而抑制病毒增殖。
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DNA, d(P-thio)(G-C-G-T-T-T-G-C-T-C-T-T-C-T-T-C-T-T-G-C-G), eicosasodium salt
DNA, d(P-thio)(G-C-G-T-T-T-G-C-T-C-T-T-C-T-T-C-T-T-G-C-G), eicosasodium salt
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- HY-108764
-
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Mipomersen sodium
|
629167-92-6 |
|
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Mipomersen sodium (ISIS 301012) 是载脂蛋白 B (apoB) 的反义寡核苷酸抑制剂。Mipomersen 具有抗 HCV 的作用，可降低 HCV 的感染性。Mipomersen sodium 可用于纯合子家族性高胆固醇血症 (HoFH) 的研究。
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DNA, d(P-thio)([2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-A-G-T-m5C-T-G-m5C-T-T-m5C-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC), sodium salt (1:19)
DNA, d(P-thio)([2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-A-G-T-m5C-T-G-m5C-T-T-m5C-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC), sodium salt (1:19)
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- HY-112980A
-
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Nusinersen sodium
|
|
99.10% |
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Nusinersen sodium 是一种反义寡核苷酸活性分子。Nusinersen sodium 修饰 SMN2 基因的前信使 RNA 剪接，从而促进全长 SMN 蛋白的生成。Nusinersen sodium 改善脊髓性肌萎缩症。
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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), sodium salt
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), sodium salt
-
- HY-139290
-
|
RGLS4326
|
2229964-07-0 |
99.06% |
|
RGLS4326 (RG4326) 是一种首创的 microRNA-17 (miR-17) 寡核苷酸抑制剂。RGLS4326 可用于常染色体显性多囊肾病 (ADPKD) 的研究。RGLS4326 作用于 HeLa 细胞，抑制 miR-17 功能，EC50 值为 28.3 nM。
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5’-AsGsCmAfCfUfUmUsGs-3‘
5’-AsGsCmAfCfUfUmUsGs-3‘
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- HY-112980
-
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Nusinersen
|
1258984-36-9 |
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Nusinersen 是一种反义寡核苷酸活性分子。Nusinersen 修饰 SMN2 基因的前信使 RNA 剪接，从而促进全长 SMN 蛋白的生成。Nusinersen 改善脊髓性肌萎缩症。
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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)
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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- HY-132581C
-
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Scrambled Tadnersen sodium
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Scrambled Tadnersen sodium 是 Tadnersen sodium (HY-132581A) 阴性对照。Tadnersen sodium，一种反义寡核苷酸 (ASO)，选择性靶向携带扩增的 C9ORF72 转录变体 1 和 3。
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Scrambled Tadnersen (sodium)
Scrambled Tadnersen (sodium)
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- HY-177453
-
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Basivarsen
|
3028750-61-7 |
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Basivarsen (DYNE-101 oligomer) 由反义寡核苷酸和连接子 (HY-177454) 组成，靶向突变型 I 型肌营养不良蛋白激酶 (DMPK) 的 RNA 进行 RHase H 介导的降解，用于肌营养不良症 1 型 (DM1) 的研究。
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DNA, d(P-thio)((2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-G-C-C-C-A-C-C-A-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)rA), 5′-[6-[[[[4-[[N-[3-[2-[2-[2-[(5aS,6R,6aR)-6-(18-carboxy-3-oxo-2,7,10,13,16-pentaoxa-4-azaoctadec-1-yl)-5,5a,6,6a,7,8-hexahydrocyclopropa[5,6]cycloocta[1,2-d]-1,2,3-triazol-1(4H)-yl]ethoxy]ethoxy]ethoxy]-1-oxopropyl]-L-valyl-N5(aminocarbonyl)-L-ornithyl]amino]phenyl]methoxy]carbonyl]amino]hexyl hydrogen phosphate]
DNA, d(P-thio)((2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-G-C-C-C-A-C-C-A-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rU-(2′-O,4′-C-methylene)m5rC-(2′-O,4′-C-methylene)rA), 5′-[6-[[[[4-[[N-[3-[2-[2-[2-[(5aS,6R,6aR)-6-(18-carboxy-3-oxo-2,7,10,13,16-pentaoxa-4-azaoctadec-1-yl)-5,5a,6,6a,7,8-hexahydrocyclopropa[5,6]cycloocta[1,2-d]-1,2,3-triazol-1(4H)-yl]ethoxy]ethoxy]ethoxy]-1-oxopropyl]-L-valyl-N5(aminocarbonyl)-L-ornithyl]amino]phenyl]methoxy]carbonyl]amino]hexyl hydrogen phosphate]
-
- HY-177484
-
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Rostudirsen
|
3028750-59-3 |
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Rostudirsen 是一种寡核苷酸， Zeleciment rostudirsen (HY-177485) 的组成部分之一。 Zeleciment rostudirsen 由 Rostudirsen (HY-177484) 与片段抗体 Zeleciment (HY-P990780) 结合而成。Zeleciment 能够与肌肉中高度表达的转铁蛋白受体 1 (TfR1) 结合。Zeleciment rostudirsen 可靶向肌肉组织，并促进细胞内51 号外显子跳跃，使肌肉细胞能够生成接近完整长度的肌营养不良蛋白。
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RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→5′)(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5′-[P-[4-[4-[(2-amino-2-oxoethyl)methylamino]-6-[[6-[[[[4-[[N-[3-[2-[2-[2-[(5aS,6R,6aR)-6-(18-carboxy-3-oxo-2,7,10,13,16-pentaoxa-4-azaoctadec-1-yl)-5,5a,6,6a,7,8-hexahydrocyclopropa[5,6]cycloocta[1,2-d]-1,2,3-triazol-1(4H)-yl]ethoxy]ethoxy]ethoxy]-1-oxopropyl]-L-valyl-N5(aminocarbonyl)-L-ornithyl]amino]phenyl]methoxy]carbonyl]amino]hexyl]oxy]-1,3,5-triazin-2-yl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2′a→5′)(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5′-[P-[4-[4-[(2-amino-2-oxoethyl)methylamino]-6-[[6-[[[[4-[[N-[3-[2-[2-[2-[(5aS,6R,6aR)-6-(18-carboxy-3-oxo-2,7,10,13,16-pentaoxa-4-azaoctadec-1-yl)-5,5a,6,6a,7,8-hexahydrocyclopropa[5,6]cycloocta[1,2-d]-1,2,3-triazol-1(4H)-yl]ethoxy]ethoxy]ethoxy]-1-oxopropyl]-L-valyl-N5(aminocarbonyl)-L-ornithyl]amino]phenyl]methoxy]carbonyl]amino]hexyl]oxy]-1,3,5-triazin-2-yl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
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- HY-132608
-
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Inotersen sodium
|
1432726-13-0 |
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Inotersen (ISIS-420915) sodium 是一种 2'-O-甲氧基乙基修饰的反义寡核苷酸。Inotersen sodium 通过靶向 TTR RNA 转录物并降低 TTR 转录物的水平来抑制转甲状腺素蛋白 (TTR) 蛋白的产生。Inotersen sodium 可用于遗传性 TTR 淀粉样变性多发性神经病的研究。
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DNA, d(P-thio)([2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-[2'- O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rG-G-T-T-A-m5C-A-T-G-A-A-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC) nonadeca sodium salt
DNA, d(P-thio)([2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-[2'- O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rG-G-T-T-A-m5C-A-T-G-A-A-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]m5rC) nonadeca sodium salt
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- HY-132580A
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Tofersen sodium
|
1898254-60-8 |
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Tofersen sodium 是一种反义寡核苷酸，可介导超氧化物歧化酶 1 (SOD1) mRNA 的 RNase H 依赖性降解，以减少 SOD1 蛋白的合成。Tofersen sodium 可用于肌萎缩侧索硬化 (ALS) 的研究。
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DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU), sodium salt
DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU), sodium salt
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- HY-132580
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Tofersen
|
2088232-70-4 |
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Tofersen (BIIB067) 是一种反义寡核苷酸，可介导超氧化物歧化酶 1 (SOD1) mRNA 的 RNase H 依赖性降解，以减少 SOD1 蛋白的合成。Tofersen 可用于肌萎缩侧索硬化 (ALS) 的研究。
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DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU)
DNA, d([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2- methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]rA-sp-Tsp-A-sp-m5C-sp-A-sp-T-sp-T-sp-T-sp-m5C-sp-T-sp-A-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-sp-[2'-O-(2-methoxyethyl)]rG-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU)
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- HY-145727A
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Volanesorsen sodium
|
1573402-50-2 |
98.51% |
|
Volanesorsen (ISIS 304801) sodium 是一种载脂蛋白CIII (apo-CIII) mRNA 的反义寡核苷酸抑制剂，可以降低甘油三酯水平并改善胰岛素抵抗。Volanesorsen sodium 可用于高甘油三酯血症，家族性乳糜小铁血症综合征和 2 型糖尿病的研究。
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DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-m5C-T-T-G-T-m5C-m5C-A-G-m5C-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU), sodium salt
DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-m5C-T-T-G-T-m5C-m5C-A-G-m5C-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU), sodium salt
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- HY-147217A
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Bepirovirsen sodium
|
2563929-84-8 |
98.44% |
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Bepirovirsen sodium 是一种反义寡核苷酸，靶向所有 HBV 信使 RNAs。Bepirovirsen sodium 导致 HBV 来源的 RNAs、HBV DNA 和病毒蛋白的减少。Bepirovirsen sodium 可用于慢性 HBV 感染的研究。Bepirovirsen sodium 结合位点序列 (GCACTTCGCTTCACCTCTGC)。
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DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rA-G-G-T-G-A-A-G-m5C-G-A-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC), sodium salt
DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rA-G-G-T-G-A-A-G-m5C-G-A-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC), sodium salt
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- HY-132586A
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Viltolarsen sodium
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Viltolarsen (NS-065/NCNP-01) sodium 是一种磷酸二酰胺吗啉代反义寡核苷酸。Viltolarsen sodium 与肌营养不良蛋白 mRNA 前体的外显子 53 结合，并通过跳过外显子 53 来恢复氨基酸开放阅读框，从而产生包含重要功能部分的缩短的肌营养不良蛋白。Viltolarsen sodium 具有用于杜氏肌营养不良症 (DMD) 研究的潜力。
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RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C), sodium salt
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-Gm5U-m5U-C), sodium salt
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- HY-145727
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Volanesorsen
|
915430-78-3 |
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Volanesorsen (ISIS 304801) 是一种载脂蛋白CIII (apo-CIII) mRNA 的反义寡核苷酸抑制剂，可以降低甘油三酯水平并改善胰岛素抵抗。Volanesorsen 可用于高甘油三酯血症，家族性乳糜小铁血症综合征和 2 型糖尿病的研究。
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DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-m5C-T-T-G-T-m5C-m5C-A-G-m5C-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)
DNA, d(P-thio)([2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-m5C-T-T-G-T-m5C-m5C-A-G-m5C-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)
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- HY-108753
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Eteplirsen
依特立生; 依特普森
|
1173755-55-9 |
98.50% |
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Eteplirsen (AVI 4658) 是一种合成的反义寡核苷酸。Eteplirsen 可用于杜氏肌营养不良症的研究。
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RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a→5')(C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), 5'-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]
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- HY-148410A
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Zorevunersen sodium
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2415330-05-9 |
98.25% |
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Zorevunersen sodium 是一种反义寡核苷酸，通过提高 SCN1A mRNA 水平从而增强钠通道 Nav1.1 蛋白的表达。Zorevunersen sodium 用于 Dravet 综合征的研究。
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RNA, (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5U-m5U-G-G-A-G-m5C-A-A-G-A-m5U-m5U-A-m5U-m5C), sodium salt
RNA, (P-thio)[2′-O-(2-methoxyethyl)](A-G-m5U-m5U-G-G-A-G-m5C-A-A-G-A-m5U-m5U-A-m5U-m5C), sodium salt
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- HY-147410
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Ulefnersen
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2589926-25-8 |
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Ulefnersen (ION363) 是一种针对肉瘤融合蛋白 (FUS) 转录本第 6 个内含子的反义寡核苷酸 Antisense Oligonucleotide (ASO) ，以非等位基因特异性方式沉默 FUS。Ulefnersen 可以在 ALS-FUS 疾病相关小鼠模型中降低大脑和脊髓中 FUS 蛋白的出生后水平，从而延缓运动神经元变性。Ulefnersen 可用于肌萎缩侧索硬化 (ALS) 的研究。
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DNA, d([2′-O-(2-methoxyethyl)]rG-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-G-sp-T-sp-m5C-sp-A-sp-m5C-sp-m5C-sp-T-sp-T-sp-T-sp-m5C-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC)
DNA, d([2′-O-(2-methoxyethyl)]rG-sp-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-G-sp-T-sp-m5C-sp-A-sp-m5C-sp-m5C-sp-T-sp-T-sp-T-sp-m5C-sp-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]rA-sp-[2′-O-(2-methoxyethyl)]m5rC-sp-[2′-O-(2-methoxyethyl)]m5rC)
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- HY-149906
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DNA, d(P-thio)(C-T-C-T-C-G-C-A-C-C-C-A-T-C-T-C-T-C-T-C-C-T-T-C-T)
DNA, d(P-thio)(C-T-C-T-C-G-C-A-C-C-C-A-T-C-T-C-T-C-T-C-C-T-T-C-T)
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- HY-145721A
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Mongersen sodium
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1443994-98-6 |
99.61% |
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Mongersen (GED-0301) sodium 是一种具有特异性和口服活性的 SMAD7 反义寡核苷酸。Mongersen sodium 恢复 TGF-β1 活性，从而抑制炎症信号。Mongersen sodium 可以减轻小鼠的克罗恩病样实验性结肠炎。
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DNA, d(P-thio)(G-T-m5C-G-C-C-C-C-T-T-C-T-C-C-C-m5C-G-C-A-G-C), sodium salt
DNA, d(P-thio)(G-T-m5C-G-C-C-C-C-T-T-C-T-C-C-C-m5C-G-C-A-G-C), sodium salt
