ReviewNature2021
The NIH Somatic Cell Genome Editing program.
Open access · hybridAbstract readReview
In one paragraph
Review in Nature, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 78 papers.
0numbers the graph read from it
0cells of the map it votes in
78citing papers in PubMed
8.5field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
78 citing papers in PubMed, 125 citations in OpenAlex.
- Combining modeling approaches for rapid preclinical evaluation of retinal gene therapy.Molecular therapy. Advances · 2026Article
- The Case for Master Protocols for Rare Neurological Diseases.Annals of neurology · 2026Review
- Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency.Nature communications · 2026Article
- Monitoring Fetal Somatic Cell Genome EditingHuman gene therapy · 2026Article
- Swine reporter model for preclinical evaluation and characterization of gene delivery vectors.Molecular therapy. Advances · 2026Article
- Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo.bioRxiv : the preprint server for biology · 2026Article
- Monitoring biological effects of somatic cell genome editing.Nature reviews. Genetics · 2026Review
- Rally around the therapeutic hub.Nature biotechnology · 2026Article
- Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys (Human gene therapy · 2026Article
- Measurement and clinical interpretation of CRISPR off-targets.Nature genetics · 2026Review
- Brain-wide Genome Editing via STEP-RNPs for Treatment of Angelman Syndrome.bioRxiv : the preprint server for biology · 2025Article
- Lipid nanoparticle-mediated mRNA delivery to CD34Nature biotechnology · 2025Article
- Alveolar Epithelial Cell Dysfunction in Acute Respiratory Distress Syndrome: Mechanistic Insights and Targeted Interventions.Biomedicines · 2025Review
- "Snip, snip, cure"? Philosophical, legal and biomedical perspectives on novel somatic genomic therapies.Medicine, health care, and philosophy · 2025Article
- Epithelial competition determines gene therapy potential to suppress Fanconi Anemia oral cancer risk.PLoS computational biology · 2025Article
- CRISPR and gene editing for kidney diseases: where are we?Clinical kidney journal · 2025Review
- Epithelial competition determines gene therapy potential to suppress Fanconi Anemia oral cancer risk.bioRxiv : the preprint server for biology · 2025Article
- Building Better Medicine: Translational Justice and the Quest for Equity in US Healthcare.The American journal of bioethics : AJOB · 2025Article
- Base editing as a therapeutic strategy for somatic repeat expansion diseases.Nature genetics · 2025Article
- The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.Nature reviews. Drug discovery · 2025Review
18 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
76 authors at 20 institutions in 2 countries.
Krishanu Saha *Department of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA. ksaha@wisc.edu.ORCID http://orcid.org/0000-0003-2837-0858
Erik J Sontheimer *RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. erik.sontheimer@umassmed.edu.ORCID http://orcid.org/0000-0002-0881-0310
P J BrooksOffice of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, MD, USA.
Melinda R DwinellDepartment of Physiology, Medical College of Wisconsin, Milwaukee, WI, USA.
Charles A GersbachDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.ORCID http://orcid.org/0000-0003-1478-4013
David R LiuMerkin Institute of Transformative Technologies, Broad Institute of MIT and Harvard, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-9943-7557
Stephen A MurrayThe Jackson Laboratory, Bar Harbor, ME, USA.
Shengdar Q TsaiDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0001-9161-3993
Ross C WilsonInnovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.ORCID http://orcid.org/0000-0002-0644-5540
Daniel G AndersonDepartment of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.ORCID http://orcid.org/0000-0001-5629-4798
Aravind AsokanDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.ORCID http://orcid.org/0000-0001-5563-4877
Jillian F BanfieldInnovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.ORCID http://orcid.org/0000-0001-8203-8771
Krystof S BankiewiczDepartment of Neurological Surgery, Ohio State University, Columbus, OH, USA.
Gang BaoDepartment of Bioengineering, Rice University, Houston, TX, USA.
Jeff W M BulteRussell H. Morgan Department of Radiology and Radiological Science, Division of MR Research, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Nenad BursacDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Jarryd M CampbellRecombinetics, Inc, Eagan, MN, USA.
Daniel F CarlsonRecombinetics, Inc, Eagan, MN, USA.ORCID http://orcid.org/0000-0001-6095-2108
Elliot L ChaikofDepartment of Surgery, Beth Israel Deaconess Medical Center, Boston, MA, USA.ORCID http://orcid.org/0000-0001-6184-4571
Zheng-Yi ChenDepartment of Otolaryngology-Head and Neck Surgery, Harvard Medical School, Boston, MA, USA.ORCID http://orcid.org/0000-0002-1452-4193
R Holland ChengDepartment of Molecular and Cellular Biology, University of California, Davis, Davis, CA, USA.
Karl J ClarkDepartment of Biochemistry and Molecular Biology, Mayo Clinic Rochester, Rochester, MN, USA.ORCID http://orcid.org/0000-0002-9637-0967
David T CurielDepartment of Radiation Oncology, Washington University in St Louis, St Louis, MO, USA.
James E DahlmanWallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA.
Benjamin E DevermanStanley Center for Psychiatric Research, Broad Institute, Cambridge, MA, USA.
Mary E DickinsonDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX, USA.
Jennifer A DoudnaInnovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
Stephen C EkkerDepartment of Biochemistry and Molecular Biology, Mayo Clinic Rochester, Rochester, MN, USA.ORCID http://orcid.org/0000-0003-0726-4212
Marina E EmborgDepartment of Medical Physics, University of Wisconsin-Madison, Madison, WI, USA.ORCID http://orcid.org/0000-0002-9351-6641
Guoping FengMcGovern Institute for Brain Research, Massachusetts Institute of Technology, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-8021-277X
Benjamin S FreedmanDivision of Nephrology, University of Washington, Seattle, WA, USA.
David M GammMcPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA.ORCID http://orcid.org/0000-0002-8152-613X
Guangping GaoHorae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0003-0097-9012
Ionita C GhiranDepartment of Medicine, Beth Israel Deaconess Medical Center, Boston, MA, USA.
Peter M GlazerDepartment of Therapeutic Radiology, Yale University, New Haven, CT, USA.ORCID http://orcid.org/0000-0003-4525-5560
Shaoqin GongDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA.
Jason D HeaneyDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.ORCID http://orcid.org/0000-0001-8475-8828
Jon D HenneboldDivision of Reproductive and Developmental Sciences, Oregon National Primate Research Center, Oregon Health and Science University, Beaverton, OR, USA.
John T HinsonPat and Jim Calhoun Cardiology Center, University of Connecticut School of Medicine, Farmington, CT, USA.
Anastasia KhvorovaRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0001-6928-8071
Samira KianiPittsburgh Liver Research Center, Department of Pathology, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
William R LagorDepartment of Molecular Physiology and Biophysics, Baylor College of Medicine, Houston, TX, USA.
Kit S LamDepartment of Biochemistry and Molecular Medicine, University of California, Davis, Davis, CA, USA.
Kam W LeongDepartment of Biomedical Engineering, Columbia University, New York, NY, USA.ORCID http://orcid.org/0000-0002-8133-4955
Jon E LevineWisconsin National Primate Research Center, University of Wisconsin-Madison, Madison, WI, USA.
Jennifer A LewisWyss Institute, Harvard University, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-0280-2774
Cathleen M LutzThe Jackson Laboratory, Bar Harbor, ME, USA.
Danith H LyDepartment of Chemistry, Carnegie-Mellon University, Pittsburgh, PA, USA.
Samantha MaraghBiomarker and Genomic Sciences Group, National Institute of Standards and Technology, Gaithersburg, MD, USA.
Paul B McCrayDepartment of Pediatrics, University of Iowa, Iowa City, IA, USA.ORCID http://orcid.org/0000-0002-4067-577X
Todd C McDevittGladstone Institute of Cardiovascular Disease, Gladstone Institutes, San Francisco, CA, USA.ORCID http://orcid.org/0000-0002-8905-4931
Oleg MirochnitchenkoOffice of Research Infrastructure Programs, Division of Program Coordination, Planning, and Strategic Initiatives, Office of the Director, National Institutes of Health, Bethesda, MD, USA.
Ryuji MorizaneDepartment of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.
Niren MurthyInnovative Genomics Institute, University of California, Berkeley, Berkeley, CA, USA.
Randall S PratherDivision of Animal Sciences, University of Missouri, Columbia, MO, USA.ORCID http://orcid.org/0000-0002-6012-4035
John A RonaldRobarts Research Institute and Department of Medical Biophysics, The University of Western Ontario, London, Ontario, Canada.
Subhojit RoyDepartment of Pathology, University of California, San Diego, La Jolla, CA, USA.
Sushmita RoyDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA.
Venkata SabbisettiDepartment of Medicine, Brigham and Women's Hospital, Boston, MA, USA.ORCID http://orcid.org/0000-0001-8696-2726
W Mark SaltzmanDepartment of Biomedical Engineering, Yale University, New Haven, CT, USA.ORCID http://orcid.org/0000-0002-2163-549X
Philip J SantangeloWallace H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology, Atlanta, GA, USA.ORCID http://orcid.org/0000-0001-7352-0339
David J SegalDepartment of Biochemistry and Molecular Medicine, University of California, Davis, Davis, CA, USA.ORCID http://orcid.org/0000-0001-8962-3105
Mary ShimoyamaDepartment of Biomedical Engineering, Marquette University and Medical College of Wisconsin, Milwaukee, WI, USA.
Melissa C SkalaDepartment of Biomedical Engineering, University of Wisconsin-Madison, Madison, WI, USA.ORCID http://orcid.org/0000-0002-6320-7637
Alice F TarantalDepartment of Pediatrics, University of California, Davis, Davis, CA, USA.
John C TiltonDepartment of Nutrition, Case Western Reserve University, Cleveland, OH, USA.
George A TruskeyDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Moriel VandsburgerDepartment of Bioengineering, University of California, Berkeley, Berkeley, CA, USA.
Jonathan K WattsRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Kevin D WellsDivision of Animal Sciences, University of Missouri, Columbia, MO, USA.
Scot A WolfeDepartment of Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, Worcester, MA, USA.ORCID http://orcid.org/0000-0002-7042-201X
Qiaobing XuDepartment of Biomedical Engineering, Tufts University, Medford, MA, USA.
Wen XueRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.
Guohua YiDepartment of Pulmonary Immunology, University of Texas Health Sciences Center at Tyler, Tyler, TX, USA.
Jiangbing ZhouDepartment of Neurosurgery, Yale University, New Haven, CT, USA.ORCID http://orcid.org/0000-0002-1201-5360
SCGE Consortium
University of Wisconsin–Madison · USUniversity of Massachusetts Chan Medical School · USDuke University · USUniversity of California, Davis · USBaylor College of Medicine · USYale University · USBeth Israel Deaconess Medical Center · USBroad Institute · USGeorgia Institute of Technology · USHarvard University · USInnovative Genomics Institute · USJackson Laboratory · USMayo Clinic · USNational Institutes of Health · USRecombinetics (United States) · USUniversity of Missouri · USBrigham and Women's Hospital · USCarnegie Mellon University · USCase Western Reserve University · USColumbia University · US
Funding
National Institute on Aging (NIA) ColonyP51OD011107 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI Simon J. Atkinson · 2012 to 2026
$191.5MWNPRC Supplemental Request for Nonhuman Primate Enclosures to Equip HIV/AIDS-Related Research FacilitiesP51OD011106 · OD · UNIVERSITY OF WISCONSIN-MADISON · PI Dorota A. Grejner-Brzezinska · 2012 to 2026
$150.7MPulmonary Toxicology Facility CoreP30ES005605 · NIEHS · UNIVERSITY OF IOWA · PI Jong Sung Kim · 1990 to 2026
$40.5MDissemination and Coordinating Center for the SCGE ConsortiumU24HG010423 · NHGRI · MEDICAL COLLEGE OF WISCONSIN · PI DWINELL, MELINDA R · 2019 to 2023
$15.0MSCGE Comparative Studies SupplementU42OD027094 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI SEGAL, DAVID J · 2019 to 2023
$14.2MPathology CoreP01HL152960 · NHLBI · UNIVERSITY OF IOWA · PI WELSH, MICHAEL J. · 2020 to 2024
$11.6MSwine Somatic Cell Genome Editing (SCGE) CenterU42OD027090 · OD · UNIVERSITY OF MISSOURI-COLUMBIA · PI PRATHER, RANDALL S, WELLS, KEVIN DALE · 2019 to 2023
$9.3MOligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7MNext-generation antisense therapeutics for ALS and frontotemporal dementiaR01NS111990 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Robert H Brown, Jonathan K Watts · 2019 to 2026
$5.2MThe Jackson Laboratory Gene Editing Testing Center (JAX-GETC)U42OD026635 · OD · JACKSON LABORATORY · PI LUTZ, CATHLEEN M, MURRAY, STEPHEN A · 2018 to 2022
$4.6MAssembly of Novel Gene Editing Particles to Understand Genome Surgery in Patient-Derived CellsR35GM119644 · NIGMS · UNIVERSITY OF WISCONSIN-MADISON · PI SAHA, KRISHANU · 2016 to 2025
$4.0MEx Vivo Generation of Functional Kidney Tissues for TransplantationUC2DK126023 · NIDDK · HARVARD UNIVERSITY · PI LEWIS, JENNIFER A. · 2020 to 2024
$4.0MNCATS NIH HHS UG3 TR002668NCATS NIH HHS UH3 TR002668NEI NIH HHS U01 EY032333NHGRI NIH HHS U24 HG010423NHLBI NIH HHS P01 HL152960NHLBI NIH HHS U01 HL145792NHLBI NIH HHS UG3 HL147352NHLBI NIH HHS UG3 HL151545NHLBI NIH HHS UH3 HL147352NHLBI NIH HHS UH3 HL147366NIAID NIH HHS U01 AI145965NIAID NIH HHS UG3 AI150550NIAID NIH HHS UG3 AI150551NIBIB NIH HHS U01 EB028892NIBIB NIH HHS U01 EB028899NIBIB NIH HHS U01 EB029371NIBIB NIH HHS UH2 EB028907NIBIB NIH HHS UH2 EB028910NIBIB NIH HHS UH3 EB028907NIBIB NIH HHS UH3 EB028910NIDDK NIH HHS DP2 DK133821NIDDK NIH HHS R56 DK122380NIDDK NIH HHS U01 DK127553NIDDK NIH HHS U01 DK127587NIDDK NIH HHS UC2 DK126023NIEHS NIH HHS P30 ES005605NIEHS NIH HHS U01 ES032673NIGMS NIH HHS R35 GM119644NIH HHS P51 OD011106NIH HHS P51 OD011107NIH HHS S10 OD020012NIH HHS S10 OD028713NIH HHS U42 OD026635NIH HHS U42 OD026645NIH HHS U42 OD027090NIH HHS U42 OD027094NINDS NIH HHS R01 NS076991NINDS NIH HHS R01 NS111990NINDS NIH HHS UG3 NS111688NINDS NIH HHS UG3 NS111689NINDS NIH HHS UH3 NS111688NINDS NIH HHS UH3 NS111689
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.
Indexed as
AnimalsCellsGene EditingGenetic TherapyGenome, HumanGoalsHumansNational Institutes of Health (U.S.)United States
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.