In Vivo Genome Editing Approach to Disrupt Hydroxyacid Oxidase 1 for the Treatment of Primary Hyperoxaluria Type 1.

已取消 10 由 honey 发布于 2026/6/22 9:18:40

DOI:10.1177/10430342261460681
作者：Jenny A Greig, Camilo Breton, Melanie K Smith, Thomas Furmanak, Peter Bell, Peter Clark, James M Wilson
文献类型：期刊论文
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Abstract：Primary hyperoxaluria type 1 (PH1) is a rare autosomal recessive disorder that leads to kidney and liver failure. PH1 is caused by a mutation in the alanine glyoxylate aminotransferase (AGXT

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