The NIH Somatic Cell Genome Editing program

dc.citation.firstpage195
dc.citation.journalTitleNature
dc.citation.lastpage204
dc.citation.volumeNumber592
dc.contributor.authorSaha, Krishanu
dc.contributor.authorSontheimer, Erik J.
dc.contributor.authorBrooks, P.J.
dc.contributor.authorDwinell, Melinda R.
dc.contributor.authorGersbach, Charles A.
dc.contributor.authorLiu, David R.
dc.contributor.authorMurray, Stephen A.
dc.contributor.authorTsai, Shengdar Q.
dc.contributor.authorWilson, Ross C.
dc.contributor.authorAnderson, Daniel G.
dc.contributor.authorAsokan, Aravind
dc.contributor.authorBanfield, Jillian F.
dc.contributor.authorBankiewicz, Krystof S.
dc.contributor.authorBao, Gang
dc.contributor.authorBulte, Jeff W.M.
dc.contributor.authorBursac, Nenad
dc.contributor.authorCampbell, Jarryd M.
dc.contributor.authorCarlson, Daniel F.
dc.contributor.authorChaikof, Elliot L.
dc.contributor.authorChen, Zheng-Yi
dc.contributor.authorCheng, R. Holland
dc.contributor.authorClark, Karl J.
dc.contributor.authorCuriel, David T.
dc.contributor.authorDahlman, James E.
dc.contributor.authorDeverman, Benjamin E.
dc.contributor.authorDickinson, Mary E.
dc.contributor.authorDoudna, Jennifer A.
dc.contributor.authorEkker, Stephen C.
dc.contributor.authorEmborg, Marina E.
dc.contributor.authorFeng, Guoping
dc.contributor.authorFreedman, Benjamin S.
dc.contributor.authorGamm, David M.
dc.contributor.authorGao, Guangping
dc.contributor.authorGhiran, Ionita C.
dc.contributor.authorGlazer, Peter M.
dc.contributor.authorGong, Shaoqin
dc.contributor.authorHeaney, Jason D.
dc.contributor.authorHennebold, Jon D.
dc.contributor.authorHinson, John T.
dc.contributor.authorKhvorova, Anastasia
dc.contributor.authorKiani, Samira
dc.contributor.authorLagor, William R.
dc.contributor.authorLam, Kit S.
dc.contributor.authorLeong, Kam W.
dc.contributor.authorLevine, Jon E.
dc.contributor.authorLewis, Jennifer A.
dc.contributor.authorLutz, Cathleen M.
dc.contributor.authorLy, Danith H.
dc.contributor.authorMaragh, Samantha
dc.contributor.authorMcCray, Paul B.
dc.contributor.authorMcDevitt, Todd C.
dc.contributor.authorMirochnitchenko, Oleg
dc.contributor.authorMorizane, Ryuji
dc.contributor.authorMurthy, Niren
dc.contributor.authorPrather, Randall S.
dc.contributor.authorRonald, John A.
dc.contributor.authorRoy, Subhojit
dc.contributor.authorRoy, Sushmita
dc.contributor.authorSabbisetti, Venkata
dc.contributor.authorSaltzman, W. Mark
dc.contributor.authorSantangelo, Philip J.
dc.contributor.authorSegal, David J.
dc.contributor.authorShimoyama, Mary
dc.contributor.authorSkala, Melissa C.
dc.contributor.authorTarantal, Alice F.
dc.contributor.authorTilton, John C.
dc.contributor.authorTruskey, George A.
dc.contributor.authorVandsburger, Moriel
dc.contributor.authorWatts, Jonathan K.
dc.contributor.authorWells, Kevin D.
dc.contributor.authorWolfe, Scot A.
dc.contributor.authorXu, Qiaobing
dc.contributor.authorXue, Wen
dc.contributor.authorYi, Guohua
dc.contributor.authorZhou, Jiangbing
dc.date.accessioned2021-05-07T19:23:50Z
dc.date.available2021-05-07T19:23:50Z
dc.date.issued2021
dc.description.abstractThe 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.
dc.identifier.citationSaha, Krishanu, Sontheimer, Erik J., Brooks, P.J., et al.. "The NIH Somatic Cell Genome Editing program." <i>Nature,</i> 592, (2021) Springer Nature: 195-204. https://doi.org/10.1038/s41586-021-03191-1.
dc.identifier.digitals41586-021-03191-1
dc.identifier.doihttps://doi.org/10.1038/s41586-021-03191-1
dc.identifier.urihttps://hdl.handle.net/1911/110498
dc.language.isoeng
dc.publisherSpringer Nature
dc.rightsThis 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.
dc.rights.urihttps://creativecommons.org/licenses/by/4.0/
dc.titleThe NIH Somatic Cell Genome Editing program
dc.typeJournal article
dc.type.dcmiText
dc.type.publicationpublisher version
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