Viltolarsen sodium_product_DataBase

Bioactivity	Viltolarsen (NS-065/NCNP-01) sodium is a phosphorodiamidate morpholino antisense oligonucleotide. Viltolarsen sodium binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. Viltolarsen sodium has the potential for Duchenne muscular dystrophy (DMD) research[1][2].
Invitro	Viltolarsen (NS-065/NCNP-01；2 天) 在删除了外显子 45-52 的 DMD 模型细胞中诱导外显子 53 跳跃，EC50 值为 0.63 μM；在删除了外显子 48-52 的DMD 模型细胞中， EC50 值为 2.3 μM[2]。Viltolarsen (10 μM；3 天) 导致 DMD 模型细胞中表达肌营养不良蛋白[2]。 MCE has not independently confirmed the accuracy of these methods. They are for reference only. 0 --> Viltolarsen sodium 相关抗体:
Formula	C244H360N113Na21O88P20
Molar Mass	7386.42
Appearance	固体
Transport	Room temperature in continental US; may vary elsewhere.
Storage	-20°C, stored under nitrogen, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen, away from moisture)
Reference	[1]. Sohita Dhillon. Viltolarsen: First Approval. Drugs. 2020 Jul;80(10):1027-1031. [2]. Naoki Watanabe, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018 Dec 7:13:442-449.

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