Spinal muscular atrophy (SMA) is caused by genetic defects in the survival motor neuron 1 (SMN1 ) gene that lead to SMN deficiency. Different SMN‐restoring therapies substantially prolong survival and function in transgenic mice of SMA. However, these therapies do not entirely prevent muscle atrophy and restore function completely. To further improve the outcome, we explored the potential of a combinatorial therapy by modulating SMN production and muscle‐enhancing approach as a novel therapeutic strategy for SMA.

中文翻译：

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肌生长抑制素抑制联合反义寡核苷酸治疗可改善脊髓性肌萎缩症的预后。

脊髓性肌萎缩症 (SMA) 是由运动神经元存活 1 ( SMN1 ) 基因的遗传缺陷导致 SMN 缺乏引起的。不同的 SMN 恢复疗法可显着延长 SMA 转基因小鼠的存活和功能。然而，这些疗法并不能完全防止肌肉萎缩和完全恢复功能。为了进一步改善结果，我们通过调节 SMN 的产生和肌肉增强方法探索了组合疗法的潜力，作为 SMA 的新治疗策略。