1. Epigenetics
  2. Small Interfering RNA (siRNA)
  3. Givosiran

Givosiran  (Synonyms: ALN-AS1)

目录号: HY-132610 纯度: 97.90%
COA 产品使用指南

Givosiran (ALN-AS1) 是一种小干扰 RNA,靶向肝脏氨基纤维素合酶 1 (ALAS1) 信使 RNA。Givosiran 下调 ALAS1 mRNA 并阻止神经毒性 δ-氨基纤维酸和卟啉的积累。Givosiran 可用于急性间歇性卟啉症的研究。

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RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-Um-Am), 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]- 14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)A-sp-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Gm-sp-Gm-sp-Um) (1:1)

Givosiran Chemical Structure

CAS No. : 1639325-43-1

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  • 生物活性

  • 纯度 & 产品资料

  • 参考文献

生物活性

Givosiran (ALN-AS1) is a small interfering RNA that targets hepatic aminolevulinate synthase 1 (ALAS1) messenger RNA. Givosiran downregulates ALAS1 mRNA and prevents accumulation of neurotoxic δ-aminolevulinic acid and porphobilinogen levels. Givosiran can be used for the research of acute intermittent porphyria[1][2].

体外研究
(In Vitro)

Givosiran 与三价 N-乙酰半乳糖胺配体偶联,后者特异性结合脱唾液酸糖蛋白受体,从而能够靶向递送至肝细胞[2]

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Clinical Trial
分子量

16300.6 (AS: 7563.9 + SS: 8736.7)

CAS 号
性状

固体

颜色

White to off-white

Sequence

RNA, (Cm-sp-Am-sp-Gm-Am-Am-Am-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)G-Um-(2'-deoxy-2'-fluoro)C-Um-(2'-deoxy-2'-fluoro)C-Am-Um-Cm-Um-Um-Am), 3'-[[(2S,4R)-1-[29-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]- 14,14-bis[[3-[[3-[[5-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-1-oxopentyl]amino]propyl]amino]-3-oxopropoxy]methyl]-1,12,19,25-tetraoxo-16-oxa13,20,24-triazanonacos-1-yl]-4-hydroxy-2-pyrrolidinyl]methyl hydrogen phosphate], complex with RNA (Um-sp-(2'-deoxy-2'-fluoro)A-sp-(2'-deoxy-2'-fluoro)A-(2'-deoxy-2'-fluoro)G-Am-(2'-deoxy-2'-fluoro)U-Gm-(2'-deoxy-2'-fluoro)A-Gm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)A-Cm-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Um-(2'-deoxy-2'-fluoro)U-Cm-(2'-deoxy-2'-fluoro)U-Gm-sp-Gm-sp-Um) (1:1)

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

-20°C, protect from light

*In solvent : -80°C, 6 months; -20°C, 1 month (protect from light)

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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目录号:
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