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The NIH Somatic Cell Genome Editing program
Nature ( IF 42.778 ) Pub Date : 2021-04-07 , DOI: 10.1038/s41586-021-03191-1
Krishanu Saha, Erik J. Sontheimer, P. J. Brooks, Melinda R. Dwinell, Charles A. Gersbach, David R. Liu, Stephen A. Murray, Shengdar Q. Tsai, Ross C. Wilson, Daniel G. Anderson, Aravind Asokan, Jillian F. Banfield, Krystof S. Bankiewicz, Gang Bao, Jeff W. M. Bulte, Nenad Bursac, Jarryd M. Campbell, Daniel F. Carlson, Elliot L. Chaikof, Zheng-Yi Chen, R. Holland Cheng, Karl J. Clark, David T. Curiel, James E. Dahlman, Benjamin E. Deverman, Mary E. Dickinson, Jennifer A. Doudna, Stephen C. Ekker, Marina E. Emborg, Guoping Feng, Benjamin S. Freedman, David M. Gamm, Guangping Gao, Ionita C. Ghiran, Peter M. Glazer, Shaoqin Gong, Jason D. Heaney, Jon D. Hennebold, John T. Hinson, Anastasia Khvorova, Samira Kiani, William R. Lagor, Kit S. Lam, Kam W. Leong, Jon E. Levine, Jennifer A. Lewis, Cathleen M. Lutz, Danith H. Ly, Samantha Maragh, Paul B. McCray, Todd C. McDevitt, Oleg Mirochnitchenko, Ryuji Morizane, Niren Murthy, Randall S. Prather, John A. Ronald, Subhojit Roy, Sushmita Roy, Venkata Sabbisetti, W. Mark Saltzman, Philip J. Santangelo, David J. Segal, Mary Shimoyama, Melissa C. Skala, Alice F. Tarantal, John C. Tilton, George A. Truskey, Moriel Vandsburger, Jonathan K. Watts, Kevin D. Wells, Scot A. Wolfe, Qiaobing Xu, Wen Xue, Guohua Yi, Jiangbing Zhou

The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH) Somatic Cell Genome Editing (SCGE) Consortium aims to accelerate the development of safer and more-effective methods to edit the genomes of disease-relevant somatic cells in patients, even in tissues that are difficult to reach. Here we discuss the consortium’s plans to develop and benchmark approaches to induce and measure genome modifications, and to define downstream functional consequences of genome editing within human cells. Central to this effort is a rigorous and innovative approach that requires validation of the technology through third-party testing in small and large animals. New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled—along with validated datasets—into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit—and the knowledge generated by its applications—as a means to accelerate the clinical development of new therapies for a wide range of conditions.



中文翻译:

NIH体细胞基因组编辑程序

从阅读到写作人类基因组的转变为改善人类健康提供了新的机会。美国国立卫生研究院(NIH)体细胞基因组编辑(SCGE)联盟旨在加速开发更安全,更有效的方法来编辑患者中与疾病相关的体细胞的基因组,即使是在难以分离的组织中也是如此。抵达。在这里,我们讨论了联盟的计划,以开发和基准化方法来诱导和测量基因组修饰,并定义人类细胞内基因组编辑的下游功能后果。这项工作的核心是严格而创新的方法,该方法要求通过对大小动物的第三方测试来验证该技术。新的基因组编辑器,用于跟踪体内已编辑细胞的递送技术和方法,以及最新开发的动物模型和人类生物系统,连同经过验证的数据集,将被组装到SCGE工具箱中,该工具箱将广泛地传播给生物医学研究界。我们将这个工具包及其应用所产生的知识可视化,以此作为加速针对各种情况的新疗法的临床开发的手段。

更新日期:2021-04-08
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