IXA4在制备治疗肌萎缩侧索硬化症的药物中的应用
CN117731659BActive Publication Date: 2026-07-17SHANDONG UNIV
Patent Information
- Authority / Receiving Office
- CN · China
- Patent Type
- Patents(China)
- Current Assignee / Owner
- SHANDONG UNIV
- Filing Date
- 2023-12-21
- Publication Date
- 2026-07-17
AI Technical Summary
Technical Problem
Existing treatment strategies for amyotrophic lateral sclerosis (ALS) are limited, lacking effective drug targets, leading to rapid disease progression and high mortality.
Method used
The IRE1-Xbp1 pathway, an endoplasmic reticulum stress-sensing molecule, was activated using IXA4. By appropriately activating the IRE1-Xbp1 pathway, the expression of XBP1 target genes was regulated, and the expression of dipeptide repeats generated from the GGGGCC hexanucleotide repeat sequence in the intron of the TDP-43 protein and C9orf72 gene was inhibited.
Benefits of technology
It significantly reduces the amount of ALS-causing proteins, improves motor function, prolongs the lifespan of animal models, and alleviates the progression of ALS disease.
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Figure CN117731659B_ABST
Abstract
本发明属于生物医药技术领域，涉及IXA4在制备治疗肌萎缩侧索硬化症的药物中的应用。本发明通过一系列细胞和动物实验证明，采用IXA4，适当激活IRE1‑Xbp1通路可以改善ALS疾病症状，延长疾病动物存活时间。本发明在现有药物的作用机理外提供了一种新的ALS的药物靶点，建立了内质网应激因子和ALS疾病的联系纽带。
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