An mRNA-based drug aims to replace a faulty enzyme and restore metabolic function in children with propionic acidemia — with encouraging early clinical results.

中文翻译：

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mRNA 疗法治疗罕见儿童疾病

一种基于 mRNA 的药物旨在替代有缺陷的酶并恢复丙酸血症儿童的代谢功能，并取得了令人鼓舞的早期临床结果。