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调查和纠正GDF11中罕见的致病突变.

Sean T Congdon1, John Bennett1, Rhoddean Opinya1

  • 1Biology Department, University of Hartford, West Hartford, CT, USA.

HGG advances
|December 19, 2025
PubMed
概括

克里斯普尔原始编辑成功模拟并纠正了导致生长延迟的GDF11基因突变. 这种基因编辑方法通过精确修复致病变异来提供一种治疗罕见遗传疾病的新策略.

关键词:
在GDF11中,GDF11是GDF11中的一个.UDN UDN UDN UDN UDN UDN UDN UDN UDN UDN未被诊断的疾病网络.主编辑主要编辑.罕见疾病是一种罕见的疾病.

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