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：用于治疗原发性高血管尿素型1（pH1）的羟基酸氧化酶1（HAO1）基因编辑的组合物和方法

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摘要

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

机译：提供了用于编辑的组合物和方法，例如，在HaO1基因内引入双链破裂。提供了用于治疗原发性高血糖尿型1（pH1）的受试者的组合物和方法。

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公开/公告号EP3830267A1

专利类型
公开/公告日2021-06-09

原文格式PDF
申请/专利权人 INTELLIA THERAPEUTICS INC.;
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申请/专利号EP20190762240
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 EP
入库时间 2022-08-24 19:16:06

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