Viltolarsen (NS-065/NCNP-01) 是一种磷酸二酰胺吗啉反义寡核苷酸，靶向肌营养不良蛋白基因第53外显子的剪接。Viltolarsen 可用于 Duchenne 肌营养不良症 (DMD) 的研究。
货号:ajcx36532
CAS:2055732-84-6
分子式:N/A
分子量：N/A
溶解度:N/A
纯度：98%
存储：Store at -20°C
库存：现货

Background:

Viltolarsen (NS-065/NCNP-01), a phosphorodiamidate morpholino antisense oligonucleotide, targets the splicing of exon 53 in the dystrophin gene. Viltolarsen can be used for the research of the Duchenne muscular dystrophy (DMD)[1].

Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has emerged as a promising therapy for DMD. Antisense oligonucleotides (AONs) are designed to induce the skipping of exon(s), in order to restore the reading frame, and therefore, allow for dystrophin expression[1].

[1]. Dzierlega K, et al. Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy. Gene Ther. 2020;27(9):407-416.