Eteplirsen (AVI 4658) 是一种合成的反义寡核苷酸。Eteplirsen 可用于杜氏肌营养不良症的研究。
货号:ajcx36596
CAS:1173755-55-9
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:

Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide. Eteplirsen can be used for Duchenne muscular dystrophy research[1].

Eteplirsen acts to promote dystrophin production by restoring the translational reading frame of Duchenne muscular dystrophy (DMD) through specific skipping of exon 51 in defective gene variants[1].

[1]. Kenji Rowel Q Lim, et al. Eteplirsen in the treatment of Duchenne muscular dystrophy. Drug Des Devel Ther. 2017 Feb 28;11:533-545.