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Updated: Feb 6, 2026

Author Spotlight: Investigating the Mechanisms and Inducing Models of Polycystic Ovary Syndrome
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雷特综合征的疾病修饰疗法:神经病学家的综述

Debopam Samanta1

  • 1Division of Child Neurology, Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR, United States.

Frontiers in neurology
|February 5, 2026
PubMed
概括

基因疗法正在出现,用于治疗雷特综合征 (RTT),这是由MECP2突变引起的严重神经发育障碍. 神经学家是实施这些先进治疗方法的关键,这些治疗方法旨在通过恢复MeCP2表达来修改疾病.

关键词:
在 AAV AAV AAV 中.基因治疗的基因疗法神经发育障碍 神经发育障碍儿科神经学 儿科神经学精密治疗疗法精确治疗疗法

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