新品上架丨内含子突变致病的典型案例：ELP1&ELP1 IVS20+6T>C家族性自主神经功能障碍人源化模型

家族性自主神经功能障碍（FD）

与ELP基因内含子突变

靶向ELP1的FD疗法及相关动物模型

B6-hELP1小鼠成功表达人源ELP1基因

总  结

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