用于治疗肌萎缩侧索硬化症的反义序列

By designing antisense sequences targeting the C9orf72 transcript and delivering them to target cells using an AAV vector, the problem of effectively blocking the formation of C9orf72 gene RNA clusters in existing technologies has been solved, realizing potential therapeutic effects for ALS and FTD.

CN115516093BActive Publication Date: 2026-07-17ASSOC INST DE MYOLOGIE +2
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Patents(China)
Current Assignee / Owner
ASSOC INST DE MYOLOGIE
Filing Date
2021-04-09
Publication Date
2026-07-17

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Abstract

本发明涉及反义序列、包含所述反义序列的核酸构建体和载体，以及它们用于治疗C9orf72六核苷酸重复扩增相关疾病如肌萎缩侧索硬化症或额颞痴呆的用途。
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