UMMS Affiliation
RNA Therapeutics Institute; Horae Gene Therapy Center; Department of Molecular, Cell and Cancer Biology
Publication Date
2021-04-07
Document Type
Article
Disciplines
Computational Biology | Genetics | Genomics | Molecular Genetics | Therapeutics
Abstract
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.
Keywords
Targeted gene repair, Genetics research
Rights and Permissions
Copyright © The Author(s) 2021. Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons license and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this license, visit http://creativecommons.org/licenses/by/4.0/.
DOI of Published Version
10.1038/s41586-021-03191-1
Source
Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB Jr, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J; SCGE Consortium. The NIH Somatic Cell Genome Editing program. Nature. 2021 Apr;592(7853):195-204. doi: 10.1038/s41586-021-03191-1. Epub 2021 Apr 7. PMID: 33828315; PMCID: PMC8026397. Link to article on publisher's site
Journal/Book/Conference Title
Nature
Related Resources
PubMed ID
33828315
Repository Citation
Saha K, Sontheimer EJ, Gao G, Khvorova A, Watts JK, Wolfe SA. (2021). The NIH Somatic Cell Genome Editing program. Open Access Publications by UMass Chan Authors. https://doi.org/10.1038/s41586-021-03191-1. Retrieved from https://escholarship.umassmed.edu/oapubs/4702
Creative Commons License
This work is licensed under a Creative Commons Attribution 4.0 License.
Included in
Computational Biology Commons, Genetics Commons, Genomics Commons, Molecular Genetics Commons, Therapeutics Commons
Comments
Full author list omitted for brevity. For the full list of authors, see article.