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Updated: Jun 1, 2025

Direct Intraventricular Delivery of Drugs to the Rodent Central Nervous System
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在肌缩侧面硬化症和亨廷顿病中使用反感性寡核酸药物

Kaili Ou1, Qingqing Jia1, Dandan Li1

  • 1State Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Key Laboratory of Non-Human Primate Research, Key Laboratory of CNS Regeneration (Ministry of Education), Guangdong-Hongkong-Macau Institute of CNS Regeneration, Jinan University, Guangzhou, 510632, China.

Translational neurodegeneration
|January 22, 2025
PubMed
概括

反感性寡核酸 (ASO) 药物显示出治疗神经退行性疾病的前景,如肌缩侧面硬化症 (ALS) 和亨廷顿病 (HD). 虽然目前的治疗方法可以控制症状,但ASO疗法提供了针对性基因方法,用于潜在的疾病修饰.

关键词:
肌缩性侧面硬化症 (AMLS) 是一种疾病.这是一种反意义的寡核酸.临床试验临床试验是指临床试验的临床试验.亨廷顿病就是亨廷顿病.

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