Self-destructing CRISPR-constructs for targeted genome editing in the retina.

用于视网膜靶向基因组编辑的自毁 CRISPR 结构。

• 批准号: nhmrc : GNT1123329
• 负责人: A/Pr Alex Hewitt
• 金额: $ 67.99万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2017
• 资助国家: 澳大利亚
• 起止时间: 2017-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/self-destructing-crispr-editing-retina/1347335
• 关键词: Self; destructing; CRISPR; constructs; targeted

Despite the identification of specific mutations causing many inherited retinal dystrophies, all of these conditions are currently untreatable. We have established gene-editing techniques and have developed a novel mouse model, which will serve as a robust platform for testing different techniques of gene editing in the retina. No other group in the world is known to be using this platform for gene editing and our work will expedite the clinical translation of this technology.

尽管鉴定了导致许多遗传性视网膜营养不良的特定突变，但所有这些病症目前都无法治疗。我们已经建立了基因编辑技术，并开发了一种新的小鼠模型，该模型将作为测试视网膜中不同基因编辑技术的强大平台。世界上没有其他团队使用这个平台进行基因编辑，我们的工作将加快这项技术的临床转化。