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
收藏
页码：901 / 902
页数：2
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