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Nusinersen
本产品不向个人销售,仅用作科学研究,不用于任何人体实验及非科研性质的动物实验。
规格:98%
分子量:7127.30
包装与价格:
包装价格(元)
1mg电议
5mg电议
10mg电议

产品介绍

Nusinersen是一种反义寡核苷酸药物,修饰 SMN2 基因的前信使 RNA 剪接,从而促进全长SMN蛋白的生成。
货号:ajcx37334
CAS:1258984-36-9
分子式:N/A
分子量:7127.30
溶解度:N/A
纯度:98%
存储:Store at -20°C
库存:现货

Background:

Nusinersen is an antisense oligonucleotide drug that modifies pre-messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein[1].

Nusinersen is an SMN-ASO that increases SMN levels by blocking an intronic splice silencer in SMN2, thereby facilitating the exon 7 inclusion and generation of FL-SMN2 transcripts[2].

[1]. Richard S Finkel, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732.
[2]. Laura Torres-Benito, et al. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Am J Hum Genet. 2019 Jul 3;105(1):221-230.