1. Apoptosis Antibody-drug Conjugate/ADC Related
  2. Transferrin Receptor Antibody-Oligonucleotide Conjugates (AOCs)
  3. Rostudirsen

Rostudirsen  (Synonyms: DYNE-251 Oligomer)

目录号: HY-177484
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Rostudirsen 是一种寡核苷酸, Zeleciment rostudirsen (HY-177485) 的组成部分之一。 Zeleciment rostudirsenRostudirsen (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]

Rostudirsen Chemical Structure

CAS No. : 3028750-59-3

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  • 生物活性

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生物活性

Rostudirsen, one of the components of Zeleciment rostudirsen (HY-177485), is a phosphorodiamidate morpholino oligomer. Zeleciment rostudirsen consists of Rostudirsen (HY-177484) conjugated to a fragment antibody (Fab), Zeleciment (HY-P990780), which binds to the transferrin receptor 1 (TfR1) that is highly expressed in muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein.

CAS 号
运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

Please store the product under the recommended conditions in the Certificate of Analysis.

纯度 & 产品资料
参考文献
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  • Do most proteins show cross-species activity?

    Species cross-reactivity must be investigated individually for each product. Many human cytokines will produce a nice response in mouse cell lines, and many mouse proteins will show activity on human cells. Other proteins may have a lower specific activity when used in the opposite species.

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Rostudirsen
目录号:
HY-177484
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