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美国国家卫生研究院体细胞基因组编辑项目

Krishanu Saha^1,2,3,4, Erik J Sontheimer⁵, P J Brooks⁶

¹Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.

|April 8, 2021

概括

美国国家卫生研究院体细胞基因组编辑 (SCGE) 联盟正在开发更安全的基因组编辑工具和方法. 这一举措旨在通过验证基因组编辑技术来加快各种疾病的新疗法.

科学领域:

生物技术
基因组学
医学研究

背景情况:

基因组编辑有望改善人类健康.
目前的方法需要进一步开发用于临床应用,特别是难以达到的组织.

研究的目的:

加快开发更安全,更有效的体细胞基因组编辑 (SCGE) 方法.
建立基因组编辑技术的严格基准测试和验证方法.
为生物医学研究界创建一个全面的工具包.

主要方法:

开发和验证体细胞的基因组编辑工具.
在人类细胞中测量基因组修饰及其功能后果.
在动物模型 (小型和大型) 中进行第三方验证.
开发体内细胞追踪方法.

主要成果:

该联盟计划将验证的数据集,基因组编辑器,交付技术和动物模型组装成一个SCGE工具包.
该工具包将包括诱导和测量基因组修改以及评估下游影响的方法.
验证将涉及临床前模型的严格测试.

结论:

将广泛传播SCGE工具包以加速临床开发.
这项倡议旨在推进基因组编辑在各种疾病中的治疗应用.
标准化验证和可访问的工具对于将基因组编辑转化为临床实践至关重要.

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