Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

被引：192
作者：
Corey, David R. [1 ,2 ]
机构：
[1] UT Southwestern, Dept Pharmacol, Dallas, TX 75235 USA
[2] UT Southwestern, Dept Biochem, Dallas, TX 75235 USA
来源：
NATURE NEUROSCIENCE | 2017年 / 20卷 / 04期
关键词：
D O I：
10.1038/nn.4508
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
Nusinersen (Spinraza) is a recently approved drug for treating spinal muscular atrophy. Approval of nusinersen may signal new opportunities for using antisense oligonucleotides as treatments for devastating neurological diseases.
引用
收藏
页码：497 / 499
页数：3
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