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Updated: Jun 12, 2026

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致病性拉敏A突变的精确基因编辑可以纠正心脏病

Xurde M Caravia1,2, Brian Hayashi1,2, Hui Li1,2

  • 1Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390.

Proceedings of the National Academy of Sciences of the United States of America
|October 13, 2025
PubMed
概括

精确的基因编辑 (BE) 纠正了Lamin A (LMNA) 基因中引起疾病的突变,在细胞和小鼠模型中有效治疗了拉米诺病. 这种基因治疗方法对未来的人类治疗有希望.

关键词:
LMNA LMNA 在线观看心肌病性心脏病 - 心肌病性心脏病基因编辑 基因编辑

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