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Updated: Jan 10, 2026

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在体内线粒体基编辑恢复基因型和视觉功能在LHON的小鼠模型

Sanghun Kim1,2, Jieun Kim1,2, Seongkwang Cha3

  • 1BK21 Graduate Program, Department of Biomedical Sciences, Korea University College of Medicine, Seoul, Republic of Korea.

Nature communications
|November 23, 2025
PubMed
概括

研究人员开发了一种高保真度基准编辑器,以创建勒伯遗传性视神经病变 (LHON) 的第一个动物模型. 这一突破使得对LHON的基因疗法的测试成为可能,这是一种由线粒体DNA突变引起的视力丧失障碍.

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