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Materials and methods for treating amyotrophic lateral sclerosis and / or frontal temporal lobe regression

机译：治疗肌萎缩性侧索硬化和/或额颞叶消退的材料和方法

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摘要

The present application is to provide an in vitro and in vivo either side, amyotrophic lateral sclerosis (ALS) and / or therapeutic agent of patients with frontal lobe degeneration (FTLD) and method. In addition, the present invention provides editing materials and methods for modulating the expression, function, or activity of the C9ORF72 gene in a cell by genomic editing.

机译：本申请旨在提供体外和体内任一方，肌萎缩性侧索硬化症（ALS）和/或额叶变性（FTLD）患者的治疗剂和方法。另外，本发明提供了通过基因组编辑来调节细胞中C9ORF72基因的表达，功能或活性的编辑材料和方法。 展开▼

著录项

公开/公告号KR20180118111A

专利类型

公开/公告日2018-10-30

原文格式PDF

申请/专利权人 크리스퍼 테라퓨틱스 아게;
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申请/专利号KR20187021173

发明设计人 코완 차드 알버트;카바디 에이미 메다;룬드버그 안테 스벤;
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申请日2016-12-22

分类号C12N15/113;A61K48;C12N15/10;C12N15/90;

国家 KR

入库时间 2022-08-21 12:38:49

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1. 用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法 [P] . 中国专利： CN109312339B . 2022.01.28

2. 用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法 [P] . 中国专利： CN109312339A . 2019-02-05

3. MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION [P] . US2021260219A1 . 2021-08-26

机译：用于治疗肌营养侧面硬化和/或前颞叶变性的材料和方法

4. MATERIALS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND/OR FRONTAL TEMPORAL LOBULAR DEGENERATION. [P] . MX2018007840A . 2019-05-02

机译：治疗肌萎缩性侧索硬化和/或颞叶颞叶退化的材料和方法。

5. Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration [P] . AU2016376191A1 . 2018-07-12

机译：治疗肌萎缩性侧索硬化和/或额颞叶小叶变性的材料和方法

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