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运动神经元负责将神经系统的信号传递给肌肉纤维。在脊髓性肌萎缩症(SMA)患者中,运动神经元缺乏生存所需的一种蛋白,结果神经元逐步死亡而患者肌肉日渐萎缩。目前,这种疾病还是个不治之症。罗氏制药(p RED)、PTC Therapeutics、SMA基金会、南加州大学和哈佛大学的研究者们,合作开发了一个可以口服的小分子药物,这种药物能够改变特定m RNA的剪切,在运动神经元中恢复关键蛋白的合成。他们用这种药物对SMA小鼠模型进行治疗,成功改善了小鼠的肌肉量、运动
Motor neurons are responsible for transmitting the nervous system’s signals to muscle fibers. In patients with Spinal Muscular Atrophy (SMA), motor neurons lack a protein required for survival and as a result, neurons gradually die while the patient’s muscles become increasingly atrophic. At present, the disease is still an incurable disease. Researchers at R & D (pRED), PTC Therapeutics, the SMA Foundation, the University of Southern California and Harvard University have collaborated on the development of an orally available small molecule drug that modifies the cleavage of specific m RNA during exercise Recovery of key protein synthesis in neurons. They used this drug to treat the SMA mouse model, successfully improving the muscle mass in mice, exercise