Gene Therapy to Prevent Blindness in Patients with Congenital Aniridia

基因疗法可预防先天性无虹膜患者失明

• 批准号: 382311
• 负责人: Simpson Elizabeth M
• 金额: $ 66.04万
• 依托单位: University of British Columbia
• 依托单位国家: 加拿大
• 项目类别: Operating Grants
• 财政年份: 2018
• 资助国家: 加拿大
• 起止时间: 2018-03-01 至 2023-03-01
• 项目状态: 已结题
• 来源: https://webapps.cihr-irsc.gc.ca/decisions/p/project_details.html?applId=382311&lang=en
• 关键词: Content Analysis; Humanized Mouse Models Of Aniridia; In Vivo Raav Crispr Therapy; In Vivo Raav Gene Augmentation Therapy; Interview-Based Analysis; Intrastromal Corneal Injection; Minipromoters For Restricted Expression; Mutation Specific And Independent

Aniridia is a rare eye disorder caused by half the normal amount of the protein paired box gene 6 (PAX6). It affects both sexes and is not known to be influenced by gender. Individuals with aniridia are typically born with low vision, and because there is

无虹膜是一种罕见的眼部疾病，由蛋白质配对盒基因6（PAX6）的一半正常量引起。它影响两性，并且不知道是否受性别的影响。患有无虹膜症的人通常出生时视力低下，