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Updated: Jun 10, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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编辑治疗阿尔法-1抗素缺乏症的方法

Derek M Erion1, Leah Y Liu1, Christopher R Brown1

  • 1Korro Bio, Inc, Cambridge, MA.

Chest
|October 14, 2024
PubMed
概括

阿尔法-1抗素 (AAT) 缺乏症是一种遗传疾病,可以通过编辑RNA序列来恢复野生类型的AAT来治疗. 编辑RNA为肺和肝脏表现提供了潜在的疾病修饰疗法.

关键词:
在 AATDD 加入阿达尔 (ADAR) 是一个叫做ADAR的词.编辑DNA的编辑编辑RNA的RNA编辑对于RNA疗法来说,它是非常重要的.

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