首页> 外国专利> COMPOSITIONS AND METHODS FOR HYDROXYACID OXIDASE 1 ( HAO1) GENE EDITING FOR TREATING PRIMARY HYPEROXALURIA TYPE 1 (PH1)

COMPOSITIONS AND METHODS FOR HYDROXYACID OXIDASE 1 ( HAO1) GENE EDITING FOR TREATING PRIMARY HYPEROXALURIA TYPE 1 (PH1)

机译：羟基氧化酶1（ HAO1 ）基因编辑的组合物和方法，用于治疗原发性高氧血症1型（PH1）

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摘要

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the HAO1 gene are provided. Compositions and methods for treating subjects having primary hyperoxaluria type 1 (PH1), are provided. 展开▼

机译：提供了在 HAO1 基因内进行编辑（例如引入双链断裂）的成分和方法。提供了用于治疗具有原发性高草酸尿症1型（PH1）的受试者的组合物和方法。 展开▼

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公开/公告号WO2020028327A1

专利类型

公开/公告日2020-02-06

原文格式PDF

申请/专利权人 INTELLIA THERAPEUTICS INC.;
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申请/专利号WO2019US44080

发明设计人 DYMEK ZACHARY WILLIAM;ODATE SHOBU;MURRAY BRADLEY ANDREW;LESCARBEAU REYNALD MICHAEL;HUEBNER ANETTE;STRAPPS WALTER;HESSE SARAH BETH;
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申请日2019-07-30

分类号C12N15/113;A61K31/7088;A61P13/12;

国家 WO

入库时间 2022-08-21 11:13:33

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3. COMPOSITIONS AND METHODS FOR HYDROXYACID OXIDASE 1 (HAO1) GENE EDITING FOR TREATING PRIMARY HYPEROXALURIA TYPE 1 (PH1) [P] . EP3830267A1 . 2021-06-09

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