News Release

小鼠的信使RNA疗法

Peer-Reviewed Publication

Proceedings of the National Academy of Sciences

Arginase mRNA

image: This is a microscopic image of the liver showing arginase mRNA delivered by lipid nanoparticle as small brown areas within hepatocytes. view more 

Credit: Image courtesy of Kristine E. Burke

肝脏酶精氨酸酶的遗传缺乏会破坏尿素循环,并且导致人类的一批症状,而一项研究发现,具有遗传性精氨酸酶缺乏的小鼠在被注射了以肝为目标的携带有精氨酸酶信使RNA的纳米颗粒,结果是使尿素循环功能和氨代谢恢复以及并使寿命得到了延长。

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