The NIH Somatic Cell Genome Editing program

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 diseas...

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Published inNature (London) Vol. 592; no. 7853; pp. 195 - 204
Main Authors Saha, Krishanu, Sontheimer, Erik J., Brooks, P. J., Dwinell, Melinda R., Gersbach, Charles A., Liu, David R., Murray, Stephen A., Tsai, Shengdar Q., Wilson, Ross C., Anderson, Daniel G., Asokan, Aravind, Banfield, Jillian F., Bankiewicz, Krystof S., Bao, Gang, Bulte, Jeff W. M., Bursac, Nenad, Campbell, Jarryd M., Carlson, Daniel F., Chaikof, Elliot L., Chen, Zheng-Yi, Cheng, R. Holland, Clark, Karl J., Curiel, David T., Dahlman, James E., Deverman, Benjamin E., Dickinson, Mary E., Doudna, Jennifer A., Ekker, Stephen C., Emborg, Marina E., Feng, Guoping, Freedman, Benjamin S., Gamm, David M., Gao, Guangping, Ghiran, Ionita C., Glazer, Peter M., Gong, Shaoqin, Heaney, Jason D., Hennebold, Jon D., Hinson, John T., Khvorova, Anastasia, Kiani, Samira, Lagor, William R., Lam, Kit S., Leong, Kam W., Levine, Jon E., Lewis, Jennifer A., Lutz, Cathleen M., Ly, Danith H., Maragh, Samantha, McCray, Paul B., McDevitt, Todd C., Mirochnitchenko, Oleg, Morizane, Ryuji, Murthy, Niren, Prather, Randall S., Ronald, John A., Roy, Subhojit, Roy, Sushmita, Sabbisetti, Venkata, Saltzman, W. Mark, Santangelo, Philip J., Segal, David J., Shimoyama, Mary, Skala, Melissa C., Tarantal, Alice F., Tilton, John C., Truskey, George A., Vandsburger, Moriel, Watts, Jonathan K., Wells, Kevin D., Wolfe, Scot A., Xu, Qiaobing, Xue, Wen, Yi, Guohua, Zhou, Jiangbing
Format Journal Article
LanguageEnglish
Published London Nature Publishing Group UK 08.04.2021
Nature Publishing Group
Subjects
13/100
13/107
42/100
42/44
45/41
59/57
59/78
631/61/201/2110
64/60
692/308/2056
Animal models
Animals
BASIC BIOLOGICAL SCIENCES
Biomedical materials
Cells - metabolism
Consortia
CRISPR
Deoxyribonucleic acid
DNA
Editing
Enzymes
Gene Editing - methods
Gene therapy
Genetic Therapy
Genome editing
Genome, Human - genetics
Genomes
Goals
Humanities and Social Sciences
Humans
In vivo methods and tests
Mitochondrial DNA
multidisciplinary
Mutation
National Institutes of Health (U.S.) - organization & administration
Patients
Perspective
Science
Science (multidisciplinary)
Sickle cell disease
Somatic cells
Toolkits
United States
United States
United States > US
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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. This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.
AbstractList 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.
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.
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.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.
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. This Perspective discusses how the Somatic Cell Genome Editing Consortium aims to accelerate the implementation of safe and effective genome-editing therapies in the clinic.
Author Vandsburger, Moriel
Santangelo, Philip J.
Gersbach, Charles A.
Liu, David R.
Clark, Karl J.
Anderson, Daniel G.
Cheng, R. Holland
Deverman, Benjamin E.
Doudna, Jennifer A.
Campbell, Jarryd M.
Glazer, Peter M.
Xue, Wen
Skala, Melissa C.
Roy, Subhojit
Morizane, Ryuji
Tarantal, Alice F.
Zhou, Jiangbing
Lutz, Cathleen M.
Lam, Kit S.
Prather, Randall S.
Dahlman, James E.
Heaney, Jason D.
Hennebold, Jon D.
Brooks, P. J.
Saltzman, W. Mark
Wilson, Ross C.
Tilton, John C.
Sontheimer, Erik J.
Feng, Guoping
Saha, Krishanu
McDevitt, Todd C.
Carlson, Daniel F.
Segal, David J.
Xu, Qiaobing
Yi, Guohua
Chen, Zheng-Yi
Roy, Sushmita
Dickinson, Mary E.
Chaikof, Elliot L.
Dwinell, Melinda R.
Ly, Danith H.
Levine, Jon E.
Mirochnitchenko, Oleg
Gong, Shaoqin
Shimoyama, Mary
Bankiewicz, Krystof S.
Truskey, George A.
Bao, Gang
Gao, Guangping
Wells, Kevin D.
Curiel, David T.
Hinson, John T.
Leong, Kam W.
McCray, Paul B.
Sabbisetti, Venkata
Ronald, John A.
Freedman, Benjamin S.
Kiani, Samira
Ekker, Stephen C.
Bursac, Nenad
Gamm, David M.
Murthy, Niren
Tsai, Shengdar Q.
Lewi
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Snippet The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health (NIH)...
Abstract The move from reading to writing the human genome offers new opportunities to improve human health. The United States National Institutes of Health...
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Animals
BASIC BIOLOGICAL SCIENCES
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Gene therapy
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Genome editing
Genome, Human - genetics
Genomes
Goals
Humanities and Social Sciences
Humans
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Mitochondrial DNA
multidisciplinary
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National Institutes of Health (U.S.) - organization & administration
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Science (multidisciplinary)
Sickle cell disease
Somatic cells
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United States
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Title The NIH Somatic Cell Genome Editing program
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