Maybe too much of a good thing in gene therapy

被引：5

作者：

Finkel, Richard S. ^{[1
]}

Fischbeck, Kenneth H. ^{[2
]}

机构：

[1] St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USA

[2] NINDS, Neurogenet Branch, NIH, Bldg 36,Rm 4D04, Bethesda, MD 20892 USA

来源：

NATURE NEUROSCIENCE | 2021年 / 24卷 / 07期

关键词：

D O I：

10.1038/s41593-021-00882-w

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

Gene replacement therapies have shown remarkable advances recently, including onasemnogene abeparvovec for treatment of symptomatic and presymptomatic patients with spinal muscular atrophy. A recent report by Van Alstyne and colleagues in a mouse model of spinal muscular atrophy raises concerns that such treatment can generate toxic overexpression of SMN protein.

引用

页码：901 / 902

页数：2

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