首页> 外国专利> COMPOSITIONS AND METHODS FOR TTR GENE EDITING AND TREATING ATTR AMYLOIDOSIS COMPRISING A CORTICOSTEROID OR USE THEREOF

COMPOSITIONS AND METHODS FOR TTR GENE EDITING AND TREATING ATTR AMYLOIDOSIS COMPRISING A CORTICOSTEROID OR USE THEREOF

机译：用于TTR基因编辑和治疗包含糖皮质激素或使用其的attr淀粉样变性的组合物和方法

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摘要

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene in combination with administration of a corticosteroid are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), in which a guide RNA and a corticosteroid are administered, are provided.

机译：本发明提供了在 TTR 基因内结合皮质类固醇的施用进行编辑的组合物和方法，例如在双链断裂中引入双链断裂。提供了用于治疗患有与运甲状腺素蛋白（ATTR）有关的淀粉样变性的受试者的组合物和方法，其中施用了指导RNA和皮质类固醇。 展开▼

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公开/公告号WO2020198706A1

专利类型

公开/公告日2020-10-01

原文格式PDF

申请/专利权人 INTELLIA THERAPEUTICS INC.;
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申请/专利号WO2020US25533

发明设计人 CHANG YONG;ALEXANDER SETH C.;WOOD KRISTY M.;KANJOLIA ARTI MAHENDRA PRAKASH;ODATE SHOBU;SEITZER JESSICA LYNN;LESCARBEAU REYNALD MICHAEL;STRAPPS WALTER;
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申请日2020-03-27

分类号A61K31;A61K31/713;A61P25;A61P25/28;C12N9;C12N9/22;C12N15/11;C12N15/113;

国家 WO

入库时间 2022-08-21 11:09:09

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1. 用于TTR基因编辑和治疗ATTR淀粉样变性的包括皮质类固醇的组合物和方法或其用途 [P] . 中国专利： CN113874004A . 2021-12-31

2. 用于TTR基因编辑及治疗ATTR淀粉样变性的组合物及方法 [P] . 中国专利： CN111417728A . 2020-07-14

3. Compositions and methods for TTR gene editing and treatment of ATTR amyloidosis comprising corticosteroids or use thereof [P] . KR20220004984A . 2022-01-12

机译：用于TTR基因的组合物和方法编辑和治疗包含皮质类固醇的attric类固醇或其用途

4. COMPOSITIONS AND METHODS FOR TTR GENE EDITING AND TREATING ATTR AMYLOIDOSIS [P] . 欧洲知识产权局专利： EP3688161A1 . 2020-08-05

机译：用于ttr基因编辑和治疗attr淀粉样变性的组合物和方法

5. compositions and methods for editing the ttr gene and treating attr amyloidosis [P] . BR112020005287A2 . 2020-09-24

机译： ttr基因和治疗attr淀粉样变性的药物组合物和方法

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