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COMPOSITIONS AND METHODS FOR LACTATE DEHYDROGENASE (LDHA) GENE EDITING

机译：乳酸脱氢酶（LDHA）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 LDHA gene are provided. Compositions and methods for treating subjects having hyperoxaluria are provided.

机译：提供了在 LDHA 基因内进行编辑的成分和方法，例如引入双链断裂。提供了用于治疗患有高草酸尿症的受试者的组合物和方法。 展开▼

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公开/公告号WO2020069296A1

专利类型

公开/公告日2020-04-02

原文格式PDF

申请/专利权人 INTELLIA THERAPEUTICS INC.;
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申请/专利号WO2019US53423

发明设计人 DYMEK ZACHARY WILLIAM;ODATE SHOBU;HUEBNER ANETTE;SRIDHAR SRIJANI;MURRAY BRADLEY ANDREW;STRAPPS WALTER;
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申请日2019-09-27

分类号C12N15/10;C12N9/04;C12N15/52;

国家 WO

入库时间 2022-08-21 11:12:09

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