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METHODS FOR TREATING INHERITED EYE DEFECTS

机译：治疗遗传性眼缺陷的方法

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摘要

Provided herein are methods for treating an inherited eye disease or disorder through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject's eyes in need of treatment. Also provided are vectors containing the nucleic acid molecule.

机译：本文提供了通过将核酸分子离体引入造血干细胞和祖细胞（HSPC），然后将HSPC移植到需要治疗的受试者的眼睛中来治疗遗传性眼病或病症的方法。治疗。还提供了包含核酸分子的载体。 展开▼

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公开/公告号WO2020210218A1

专利类型

公开/公告日2020-10-15

原文格式PDF

申请/专利权人 THE REGENTS OF THE UNIVERSITY OF CALIFORNIA;
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申请/专利号WO2020US27050

发明设计人 CHERQUI STEPHANIE;
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申请日2020-04-07

分类号C12N15/10;A61P27/02;C12N5/075;C12N15/63;C12N15/86;

国家 WO

入库时间 2022-08-21 11:08:55

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