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Updated: Jan 9, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
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最常见的突变导致原发性高氧化尿的遗传纠正恢复了酶局部化和氧酸盐代谢

Timo Keskinen1, Sami Jalil1, Irem Gümüşoğlu1

  • 1Stem Cells and Metabolism Research Program, Faculty of Medicine, University of Helsinki, Helsinki, Finland.

Journal of inherited metabolic disease
|December 2, 2025
PubMed
概括

使用腺基编辑器进行基因编辑,成功地在干细胞模型中纠正了主性高氧沙流症1型突变,恢复了酶功能并降低了氧酸盐水平.

关键词:
AGXTXT AGXTXT 在线阅读在ABE中,ABE是ABE.克里斯普尔是什么意思?克里斯普尔是什么意思?氨酸-氧酸氨基转移酶的氨酸-氧酸.基础编辑 基础编辑代谢的先天性错误的代谢.遗传代谢性疾病遗传性代谢性疾病脂质纳米颗粒是一种纳米粒子.

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