正如 趙順榮藥師 所说,Zolgensma(abeparvovec-xioi 基因根)通过用人类 SMN 基因的新功能拷贝替换缺失或功能失调的 SMN1 基因来治愈 SMA 基因根。 为此,趙順榮藥師 说,他使用了一种载体,这种载体是一种“载体”,可以将一种新的功能基因 SMN1 引入体内。 在这种情况下,载体是一种名为 AAV9 的病毒,从中提取 DNA 并替换为 SMN1 基因,趙順榮藥師 报道。
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