包装: | 1mg |
规格: | 92% |
市场价: | 11907元 |
分子量: | 8647.00 |
Golodirsen (SRP-4053) 是一种磷酸二亚胺的吗啉代低聚物 (PMO),特异性地靶向肌营养不良蛋白培养蛋白前 mRNA 的外显子 53。Golodirsen 可用于杜氏肌营养不良症 (DMD) 的研究。
货号:ajcx37654
CAS:1422959-91-8
分子式:N/A
分子量:8647.00
溶解度:N/A
纯度:92%
存储:Store at -20°C
库存:现货
Background:
Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) that specifically targets exon 53 of dystrophin pre-mRNA. Golodirsen can be used for the research of Duchenne muscular dystrophy (DMD)[1][2].
Golodirsen specifically targets exon 53 of dystrophin pre-mRNA, resulting in its exclusion from the final mRNA product. Skipping of exon 53 results in restoration of the mRNA reading frame and leads to the production of a partially internally deleted dystrophin protein with intact C and N-terminal regions[2].
[1]. Heo YA. Golodirsen: First Approval. Drugs. 2020 Feb;80(3):329-333.
[2]. Scaglioni D, et, al. The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy. Acta Neuropathol Commun. 2021 Jan 6;9(1):7.