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Updated: Jun 13, 2025

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对尼曼-皮克C1疾病的潜在治疗方法的研究进展

Caifeng Zhang1, Keke Su1,2, Xu Jiang1,2

  • 1Department of Gastroenterology, The First Affiliated Hospital of Xinxiang Medical University, Xinxiang, China.

Frontiers in pharmacology
|September 12, 2024
PubMed
概括

尼曼-皮克病C1型 (NP-C1) 是一种罕见的遗传疾病,影响胆固醇运输. 目前的研究探讨了各种治疗策略,包括小分子,细胞疗法和基因疗法,以对抗这种疾病.

关键词:
尼曼 - 皮克病C1型基于细胞的疗法.组合疗法治疗组合治疗.疾病模型 疾病模型基因治疗的基因疗法小分子治疗小分子疗法

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