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Updated: Jul 6, 2025

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针对线粒体疾病的基因疗法

Nandaki Keshavan1,2, Michal Minczuk3, Carlo Viscomi4,5

  • 1UCL Great Ormond Street Institute of Child Health, London, UK.

Journal of inherited metabolic disease
|January 3, 2024
PubMed
概括

基因疗法在使用腺相关病毒载体和基因编辑工具治疗原发性线粒体疾病 (PMDs) 方面表现有前途. 莱伯遗传性视神经病变的临床试验正在推进,但对器官向和试验设计的挑战仍然存在.

关键词:
在 AAV AAV AAV 中.克里斯普尔是什么意思?克里斯普尔是什么意思?伦敦LHON 在线观看基因编辑 基因编辑基因治疗的基因疗法线粒体疾病是线粒体疾病.

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