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Updated: May 30, 2025

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通过基编辑在小鼠中纠正致病性线粒体DNA突变

Jose D Barrera-Paez1, Sandra R Bacman2, Till Balla3

  • 1Graduate Program in Human Genetics, University of Miami Miller School of Medicine, 1501 NW 10th Avenue (M-860), Miami, FL 33136, USA.

Science translational medicine
|January 29, 2025
PubMed
概括

使用DddA衍生的细胞因子基编辑器 (DdCBE) 的线粒体基因治疗成功地恢复了小鼠的线粒体转移RNA (tRNA) 氨酸水平. 然而,仔细的剂量监测至关重要,以防止非目标线粒体DNA编辑和不良影响.

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