Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

Kim Jeong-Ki; Monani Umrao R.

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Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

机译：增强SMN蛋白治疗婴儿脊柱肌肉萎缩

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Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.

机译：脊柱肌肉萎缩（SMA）是由SMN蛋白不足引起的常见和致命的小儿科神经肌病。现在，两项临床试验（Mendell等，2017; Finkel等，2017）证明恢复蛋白质是治疗性的，为SMA患者提供新的治疗选择，并将希望更新。

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《Neuron》 |2018年第5期|共3页
作者
Kim Jeong-Ki; Monani Umrao R.;
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Columbia Univ Dept Pathol Med Ctr New York NY 10032 USA;

Columbia Univ Dept Pathol Med Ctr New York NY 10032 USA;

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正文语种 eng
中图分类神经病学;
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1. Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy [J] . Kim Jeong-Ki, Monani Umrao R. Neuron . 2018,第5期

机译：增强SMN蛋白治疗婴儿脊柱肌肉萎缩
2. SMN and Gemins: 'We are family'... or are we? Insights into the partnership between Gemins and the spinal muscular atrophy disease protein SMN [J] . BioEssays : . 2010,第12期

机译：SMN和Gemins：“我们是一家人” ...还是我们？洞悉Gemins与脊髓性肌萎缩症蛋白SMN之间的伙伴关系
3. Reduced survival of motor neuron (SMN) protein in motor neuronal progenitors functions cell autonomously to cause spinal muscular atrophy in model mice expressing the human centromeric (SMN2) gene. [J] . Park GH, Maeno Hikichi Y, Awano T, The Journal of Neuroscience: The Official Journal of the Society for Neuroscience . 2010,第36期

机译：运动神经元祖细胞中运动神经元（SMN）蛋白存活率的降低会自动发挥功能，导致表达人类着丝粒（SMN2）基因的模型小鼠的脊髓肌萎缩。
4. A Motor Imagery-based Brain-Computer Interface Scheme for a Spinal Muscular Atrophy Subject in CYBATHLON Race [C] . Shi-Chun Bao, Kai Yuan, Cheng Chen, International IEEE/EMBS Conference on Neural Engineering . 2021

机译：基于电机图像的脑电电脑界面方案，用于Cybathlon Race中的脊柱肌肉萎缩课程
5. The Smn-independent beneficial effects of trichostatin A on an intermediate mouse model of spinal muscular atrophy. [D] . Yazdani, Armin. 2014

机译：曲古抑菌素A对脊髓性肌萎缩症的中级小鼠模型的Smn依赖性有益作用。
6. Limited Phenotypic Effects of Selectively Augmenting the SMN Protein in the Neurons of a Mouse Model of Severe Spinal Muscular Atrophy [O] . Andrew J-H. Lee, Tomoyuki Awano, Gyu-Hwan Park, -1

机译：选择性增广的限量表型效应严重脊髓性肌萎缩症的小鼠模型中的神经元的smN蛋白
7. Limited phenotypic effects of selectively augmenting the SMN protein in the neurons of a mouse model of severe spinal muscular atrophy. [O] . Andrew J-H Lee, Tomoyuki Awano, Gyu-Hwan Park, 2012

机译：在严重脊髓性肌萎缩的小鼠模型中选择性增加smN蛋白的有限表型效应。

Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy

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