通过主要编辑成功地纠正了RYR1基因中对肌肉病负责的突变

Kelly Godbout1,2, Joël Rousseau2, Jacques P Tremblay1,2

  • 1Molecular Biology Department, Laval University, Quebec, QC G1V 0A6, Canada.

Cells
|January 11, 2024
PubMed
概括

总编辑成功地纠正了人类肌细胞中的RYR1基因突变,为RYR1相关肌病症提供了潜在的基因疗法. 这一突破证明了纠正导致肌肉衰弱的遗传缺陷的可行性.

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