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Updated: Jan 17, 2026

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用于治疗α-1抗素缺乏症的RNA编辑

Prashant Monian1, Chikdu Shivalila1, Genliang Lu1

  • 1Wave Life Sciences, Cambridge, MA 02138, United States.

Nucleic acids research
|January 14, 2026
PubMed
概括

一种新的RNA编辑疗法,SERPINA1-994,对阿尔法-1抗素缺乏症 (AATD) 有希望. 它纠正肝细胞中的Z突变,改善小鼠的肺和肝功能.

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