1. Cell Cycle/DNA Damage
  2. DNA/RNA Synthesis
  3. Nusinersen

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)

Nusinersen Chemical Structure

CAS No. : 1258984-36-9

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规格 价格 是否有货 数量
1 mg ¥3780
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MCE 顾客使用本产品发表的 1 篇科研文献

  • 生物活性

  • 纯度 & 产品资料

  • 参考文献

生物活性

Nusinersen is an antisense oligonucleotide active molecule. Nusinersen modifies the pre-messenger RNA splicing of the SMN2 gene, thereby promoting the production of full-length SMN protein. Nusinersen improves spinal muscular atrophy[1][2][3].

体外研究
(In Vitro)

Nusinersen 是一种 SMN-ASO,它通过阻断 SMN2 中的内含子剪接抑制物来提高 SMN 水平,从而促进外显子 7 的纳入和 FL-SMN2 转录本的产生[2]

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

体内研究
(In Vivo)

Nusinersen (6.5 mg/kg;脑室内注射;出生后第 1 天) 可改善 SMA 小鼠模型中的运动功能并防止运动神经元 Cajal 体分解和异常的 poly(A) RNA 分布[3]

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

Animal Model: SMNΔ7 mice (Smn-/-;SMN2+/+; SMN∆7+/+, male/female), a severe SMA mouse model[3]
Dosage: 6.5 mg/kg
Administration: Intracerebroventricular (ICV) injection; at postnatal day 1
Result: Improved righting reflex latency, preserved αMN number, reduced motor neuron degeneration, and rescued skeletal muscle fiber size and oxidative metabolism.
Increased SMN protein in spinal cord but not in muscle.
Clinical Trial
分子量

7127.30

CAS 号
性状

固体

颜色

White to off-white

Sequence

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)

运输条件

Room temperature in continental US; may vary elsewhere.

储存方式

-20°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

纯度 & 产品资料

纯度: 97.79%

参考文献
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  • 稀释计算器

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Help & FAQs
  • 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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产品名称:
Nusinersen
目录号:
HY-112980
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