Base editors can restore the expression of survival motor neuron protein to therapeutically beneficial levels in animal and cell models of spinal muscular atrophy.

中文翻译：

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脊髓性肌萎缩症的突变校正

在脊髓性肌萎缩症的动物和细胞模型中，碱基编辑器可以将存活运动神经元蛋白的表达恢复到治疗有益的水平。