2. 寡核苷酸
  3. 反义寡核苷酸
  4. Ulefnersen sodium

Ulefnersen sodium  (Synonyms: ION-363 sodium)

目录号: HY-147410A 纯度: 94.55%
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摩尔计算器

Ulefnersen sodium (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 sodium 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 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)]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), sodium salt

Ulefnersen sodium Chemical Structure

CAS No. : 2589926-27-0

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1 mg ￥1430
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5 mg ￥3910
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Ulefnersen sodium 相关抗体

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  • 生物活性

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  • 参考文献

生物活性

Ulefnersen sodium (ION363) is an Antisense Oligonucleotide (ASO) directed against the 6th intron of the fused-in sarcoma (FUS) transcript to silence FUS in a non-allele-specific manner. Ulefnersen sodium can reduce postnatal levels of FUS protein in the brain and spinal cord in disease-relevant mouse model of ALS-FUS , delaying motor neuron degeneration. Ulefnersen sodium can be used in the research of Amyotrophic Lateral Sclerosis (ALS) [1].

体内研究
(In Vivo)

Ulefnersen sodium (ION363) (脑室内注射, 20 μg, 总共一次) 有效地减少了 P517/WT mice 大脑和脊髓中的 FUS 蛋白的表达，逆转了与突变 FUS 毒性相关的 RNA 结合蛋白 (RBPs) 的去污剂不溶性[1]。
Ulefnersen sodium (脑室内注射, 20 μg, 总共一次) 有效地沉默了 P517L 和 Δ14 杂合子小鼠大脑和脊髓中的 Fus 基因以及降低了 FUS 蛋白的出生后水平，从而延缓了运动神经元的退化[1]。

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

Animal Model: Newborn (postnatal day 1 (P1)) P517/WT mice [1]
Dosage: 20 μg
Administration: Intracerebroventricular injection, Once in total
Result: Showed an overall decrease in both wild-type and mutant FUS protein to approximately 20–50% of the levels observed in matched controls at 1 month of age, as determined by western immunoblot analysis of brain and spinal cord.
Decreased the levels of TDP-43, hnRNP A1, hnRNP U and UPF1 in the detergent-insoluble fractions.
Showed strong nuclear signals in all cells, including MNs at 1 and 4 months. However, signals for both Ulefnersen and NTC ASO dropped to near background levels by 6 months, as determined by analysis of anti-ASO immunostaining.
Animal Model: Newborn (P1) MN-P517L/Δ14 mice[1]
Dosage: 20 μg
Administration: Intracerebroventricular injection, Once in total
Result: Observed no MN loss and prevented muscle denervation and microgliosis at 4 months. Observed an increase in muscle denervation and microgliosis at 6 months, indicating that the onset of disease pathology was delayed rather than completely prevented. However, observed no MN degeneration at 6 months.
分子量

7477.40

Formula

C230H302N68O126P19S13Na19
CAS 号
性状

固体

颜色

White to off-white

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
参考文献

Ulefnersen sodium 相关分类

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Keywords:

Ulefnersen2589926-27-0ION-363ION363ION 363OthersAntisense OligonucleotideASOFused-in sarcomaFUSAmyotrophic Lateral SclerosisALSNeurological diseaseInhibitorinhibitorinhibit

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