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.