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在寻找脊柱肌肉缩的疾病修饰者

Daria Chudakova1, Ludmila Kuzenkova1, Andrey Fisenko1

  • 1National Medical Research Center of Children's Health of the Ministry of Health of the Russian Federation, 119991 Moscow, Russia.

International journal of molecular sciences
|October 26, 2024
PubMed
概括

脊柱肌缩 (SMA) 是一种影响运动神经元的遗传疾病. 疾病修饰剂显著影响SMA的SMA.

关键词:
一个SMN1的SMN1在SMN2中使用SMN2.疾病修饰剂疾病修饰剂遗传性自体逆向性遗传性疾病神经退行症的神经退行症脊柱肌肉缩 脊柱肌肉缩 脊柱肌肉缩

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