Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

被引：195

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

页码：497 / 499

页数：3

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