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 (ASOs) usually refer to shortsyntheticsingle-stranded DNA or RNA (13-30 nucleotides). Following binding to the targeted mRNA or pre-mRNAASOs modulates RNA function by several different mechanisms.
1. ASOs can form an RNA–DNA hybrid that becomes a substrate for RNase Hresulting in target mRNA degradation.
2. ASOs can modulate gene expression via steric block of the ribosomal machinerywhich can lead to reduced expressionmodulation of splicing and/or restoration of a functional protein.
3. Binding of ASOs to pre-mRNA can alter splicing factor recruitment and regulate splicing events.
The first in vivo applications of ASOs showed limited clinical potential because of the high susceptibility of ASOs with an unmodified phosphoribose backbone to rapid degradation by endonucleases and exonucleases. The modifications in backboneand sugar molecules give ASOs more affinity and stability. Phosphorodiamidate morpholino oligomers (PMO) are very resistant to nuclease and protease degradation and are mostly used in splicing modulation or translation inhibition. Chemical modifications at 2' position of ribose sugar ring such as 2’-O-methyl (2’-O-me)2’-Fluoro (2’-F)2’-O-methoxyethyl (2’-MOE) allow oligonucleotide to adopt RNA like C3’-endo sugar puckermaking it thermally stable.
Antisense Oligonucleotides (600):
| Cat. No. |
Product Name |
CAS No. |
Purity |
Chemical Structure |
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- HY-109528
-
|
Fomivirsen sodium
|
160369-77-7 |
99.71% |
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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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DNAd(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
DNAd(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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DNAd(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)
DNAd(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
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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
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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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DNAd(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
DNAd(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-139290
-
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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-150364
-
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Farabursen
|
2953012-32-1 |
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Farabursen (RGLS8429) 是一种用于研究常染色体显性多囊肾病的寡核苷酸，其作用是抑制 miR-17 并优先靶向肾脏。
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RNA(P-thio)([2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]G-Cm-(2′-deoxy-2′-fluoro)A-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)U-Um-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]U-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A)
RNA(P-thio)([2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]G-Cm-(2′-deoxy-2′-fluoro)A-(2′-deoxy-2′-fluoro)C-(2′-deoxy-2′-fluoro)U-Um-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]U-(3′→4′)-[2′,5′-anhydro-6′-deoxy-4′-C-(hydroxymethyl)-α-L-mannofurano]A)
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- HY-177662
-
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Sapablursen
|
2273007-95-5 |
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Sapablursen 是一种反义寡核苷酸，可减少 TMPRSS6 的生成，从而提高血清铁调素的表达量。血清铁调素是铁稳态的关键调节因子。Sapablursen 可用于如真性红细胞增多症等血液疾病的研究中。
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DNAd([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rA-T-sp-T-sp-m5C-sp-m5C-sp-A-sp-A-sp-A-sp-G-sp-G-sp-G-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU)5'-[26-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl hydrogen phosphate]
DNAd([2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]m5rU-[2'-O-(2-methoxyethyl)]rA-T-sp-T-sp-m5C-sp-m5C-sp-A-sp-A-sp-A-sp-G-sp-G-sp-G-sp-[2'-O-(2-methoxyethyl)]m5rC-[2'-O-(2-methoxyethyl)]rA-[2'-O-(2-methoxyethyl)]rG-sp-[2'-O-(2-methoxyethyl)]m5rC-sp-[2'-O-(2-methoxyethyl)]m5rU)5'-[26-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-14,14-bis[[3-[[6-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]hexyl]amino]-3-oxopropoxy]methyl]-8,12,19-trioxo-16-oxa-7,13,20-triazahexacos-1-yl hydrogen phosphate]
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- HY-177653A
-
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DNAd(P-thio)([2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rU-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rU-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano](3'→4')m5rU-T-G-T-A-A-G-T-G-m5C-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]rA-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]rA-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rC-[2'-O-(2-methoxyethyl)]m5rC)sodium salt (1:15)
DNAd(P-thio)([2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rU-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rU-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano](3'→4')m5rU-T-G-T-A-A-G-T-G-m5C-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]rA-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]rA-(3'→4')-[2',5'-anhydro-6'-deoxy-4'-C-(hydroxymethyl)-α-L-mannofurano]m5rC-[2'-O-(2-methoxyethyl)]m5rC)sodium salt (1:15)
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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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DNAd([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)
DNAd([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-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-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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DNAd([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
DNAd([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-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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DNAd(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
DNAd(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 |
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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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DNAd(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
DNAd(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-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-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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DNAd(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)
DNAd(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
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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-148687A
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SPC5001 sodium
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98.89% |
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SPC5001 sodium 是一种锁核酸 (LNA) 修饰的反义寡核苷酸 (ASO)，与人 PCSK9 (前蛋白转化酶枯草杆菌蛋白酶/kexin 9 型) mRNA 互补。 SPC5001 sodium 可用于高胆固醇血症的研究。 SPC5001 序列：5'-TGmCTACAAAACmCmCA-3'。
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5′-TGmCTACAAAACmCmCA-3′sodium salt
5′-TGmCTACAAAACmCmCA-3′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-145722
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Apatorsen sodium
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915443-09-3 |
98.54% |
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Apatorsen (OGX-427) sodium 是一种2'-甲氧基乙基修饰的反义寡核苷酸，也是一种 Hsp27 抑制剂。Apatorsen sodium 可降低 Hsp27 mRNA和蛋白水平，削弱应激诱导的细胞保护功能，诱导细胞凋亡，抑制肿瘤生长并阻止转移。Apatorsen sodium 适用于非小细胞肺癌、去势抵抗性前列腺癌、乳腺癌、卵巢癌和膀胱癌的相关研究。
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DNAd(P-thio)([2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rA-m5C-G-m5C-G-G-m5C-G-m5C-T-m5C-G-G-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)nonadecasodium salt
DNAd(P-thio)([2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]rA-m5C-G-m5C-G-G-m5C-G-m5C-T-m5C-G-G-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)nonadecasodium salt
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- HY-143230
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Custirsen
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890056-27-6 |
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Custirsen 是一种高度特异性的反义寡核苷酸，可抑制 clusterin的产生，clusterin 是一种抗凋亡蛋白，在化疗反应中上调表达，并导致耐药性。
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DNAd(P-thio)([2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-A-G-C-A-G-A-G-T-C-T-T-C-A-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)
DNAd(P-thio)([2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]rG-[2′-O-(2-methoxyethyl)]m5rC-A-G-C-A-G-A-G-T-C-T-T-C-A-[2′-O-(2-methoxyethyl)]m5rU-[2′-O-(2-methoxyethyl)]m5rC-[2′-O-(2-methoxyethyl)]rA-[2′-O-(2-methoxyethyl)]m5rU)
