Evidence map›Paper›PMID 41530266›Full record

ReviewNature reviews. Genetics2026

Monitoring biological effects of somatic cell genome editing.

Benjamin S Freedman, Jeff W M Bulte, Bruce R Conklin, Luke M Judge, Melinda R Dwinell, Aron M Geurts, Madeleine J Sitton, Vineet Mahajan, Samira Kiani, Charles A Gersbach and 18 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Nature reviews. Genetics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Review
  5. Review
  6. Review
  7. Article
  8. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

28 authors.

Benjamin S FreedmanDepartment of Medicine, Division of Nephrology, Kidney Research Institute, and Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA. benof@uw.edu.ORCID http://orcid.org/0000-0003-2228-7383
Jeff W M BulteDepartment of Radiology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.ORCID http://orcid.org/0000-0003-1202-1610
Bruce R ConklinGladstone Institutes, San Francisco, CA, USA.
Luke M JudgeGladstone Institutes, San Francisco, CA, USA.
Melinda R DwinellDepartment of Physiology, Medical College of Wisconsin, Milwaukee, WI, USA.
Aron M GeurtsDepartment of Physiology, Medical College of Wisconsin, Milwaukee, WI, USA.
Madeleine J SittonDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Vineet MahajanDepartment of Pathology, University of Pittsburgh, Pittsburgh, PA, USA.
Samira KianiDepartment of Pathology, University of Pittsburgh, Pittsburgh, PA, USA.
Charles A GersbachDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.ORCID http://orcid.org/0000-0003-1478-4013
Mo R EbrahimkhaniDepartment of Pathology, University of Pittsburgh, Pittsburgh, PA, USA.
John J KellyRobarts Research Institute, University of Western Ontario, London, Ontario, Canada.ORCID http://orcid.org/0000-0002-3626-8629
John A RonaldRobarts Research Institute, University of Western Ontario, London, Ontario, Canada.ORCID http://orcid.org/0000-0003-3665-173X
Ryuji MorizaneDepartment of Medicine, Harvard Medical School, Boston, MA, USA.
Navin GuptaDepartment of Medicine, Mayo Clinic College of Medicine, Rochester, MN, USA.
Ali Shakeri-ZadehDepartment of Radiology and Institute for Cell Engineering, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Nicole VoDepartment of Medicine, Division of Nephrology, Kidney Research Institute, and Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
Krishanu SahaDepartments of Biomedical Engineering and Pediatrics, and McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA.ORCID http://orcid.org/0000-0003-2837-0858
Shivani SaxenaDepartments of Biomedical Engineering and Pediatrics, and McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA.
David M GammDepartment of Ophthalmology and Visual Sciences, Waisman Center, and McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA.
Divya SinhaDepartment of Ophthalmology and Visual Sciences, Waisman Center, and McPherson Eye Research Institute, University of Wisconsin-Madison, Madison, WI, USA.ORCID http://orcid.org/0000-0002-5854-0056
Alice F TarantalDepartments of Pediatrics and Cell Biology and Human Anatomy, School of Medicine, and California National Primate Research Center, University of California Davis, Davis, CA, USA.ORCID https://orcid.org/0000-0002-8153-1094
Moriel VandsburgerDepartment of Bioengineering, University of California Berkley, Berkeley, CA, USA.
Azusa MatsubaraDepartment of Hematology, St Jude's Children's Research Hospital, Memphis, TN, USA.
Hongxia FuDepartment of Medicine, Division of Nephrology, Kidney Research Institute, and Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
Shengdar Q TsaiDepartment of Hematology, St Jude's Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0001-9161-3993
SCGE Dissemination and Coordinating Center Toolkit Team
SCGE Biological Effects Initiative

Funding

National Institute on Aging (NIA) ColonyP51OD011107 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI Simon J. Atkinson · 2012 to 2026
$191.5M
Vector and Transgenic Mouse CoreP30DK017047 · NIDDK · UNIVERSITY OF WASHINGTON · PI Sakeneh Zraika · 1986 to 2026
$41.4M
The CRISPR Vision Program: Nonviral Genome Editing Platforms to Treat Inherited Retinal Channelopathies Epqt SupplementU19NS132296 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI Cathy Rasmussen · 2023 to 2026
$36.8M
SCGE Comparative Studies SupplementU42OD027094 · OD · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HARTIGAN-O'CONNOR, DENNIS J., SEGAL, DAVID J · 2019 to 2023
$14.2M
Training/Dissemination-Resource for Molecular Imaging Agents in Precision MedicineP41EB024495 · NIBIB · UT SOUTHWESTERN MEDICAL CENTER · PI MARTIN G POMPER · 2017 to 2026
$11.8M
Assembly of Novel Gene Editing Particles to Understand Genome Surgery in Patient-Derived CellsR35GM119644 · NIGMS · UNIVERSITY OF WISCONSIN-MADISON · PI SAHA, KRISHANU · 2016 to 2025
$4.0M
Recreating Kidney Organogenesis in vitro with Human Pluripotent Stem CellsDP2DK133821 · NIDDK · MASSACHUSETTS GENERAL HOSPITAL · PI MORIZANE, RYUJI · 2019 to 2019
$2.5M
JAX-Gladstone, SCGE Disease Models Studies SupplementU01ES032673 · NIEHS · J. DAVID GLADSTONE INSTITUTES · PI CONKLIN, BRUCE R · 2020 to 2022
$2.4M
Enabling Nanoplatforms for Targeted in vivo Delivery of CRISPR/Cas9 Ribonucleoproteins in the BrainUG3NS111688 · NINDS · UNIVERSITY OF WISCONSIN-MADISON · PI EMBORG, MARINA, GONG, SHAOQIN - · 2018 to 2020
$2.3M
SCGE Comparative Studies SupplementU01DK127553 · NIDDK · UNIVERSITY OF WASHINGTON · PI FREEDMAN, BENJAMIN SOLOMON · 2020 to 2022
$2.2M
Vascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editingU01DK127587 · NIDDK · MASSACHUSETTS GENERAL HOSPITAL · PI LEWIS, JENNIFER A., MORIZANE, RYUJI · 2020 to 2022
$2.1M
PET/CT for Nonhuman Primate ImagingS10RR025063 · NCRR · UNIVERSITY OF CALIFORNIA AT DAVIS · PI TARANTAL, ALICE F · 2011 to 2011
$2.1M
NCRR NIH HHS S10 RR025063NHLBI NIH HHS R01 HL130533NHLBI NIH HHS U01 HL145792NHLBI NIH HHS U01 HL156348NIAID NIH HHS U01 AI176460NIA NIH HHS R01 AG072052NIBIB NIH HHS P41 EB024495NIBIB NIH HHS U01 EB028892NIBIB NIH HHS UH2 EB028904NIBIB NIH HHS UH2 EB028907NIBIB NIH HHS UH2 EB028910NIBIB NIH HHS UH3 EB028904NIBIB NIH HHS UH3 EB028910NIDDK NIH HHS DP2 DK133821NIDDK NIH HHS P30 DK017047NIDDK NIH HHS R21 DK129909NIDDK NIH HHS U01 DK127553NIDDK NIH HHS U01 DK127587NIEHS NIH HHS U01 ES032673NIGMS NIH HHS R35 GM119644NIGMS NIH HHS R35 GM149516NIH HHS P51 OD011107NIH HHS S10 OD016261NIH HHS S10 OD018102NIH HHS S10 OD026740NIH HHS S10 OD028713NIH HHS U42 OD027094NINDS NIH HHS R01 NS119678NINDS NIH HHS U19 NS132296NINDS NIH HHS UG3 NS111688
6 · The paper itself

Abstract

CRISPR-based genome editing therapeutics are entering the clinic, offering transformative potential but also presenting potential risks. Preclinical-to-clinical toolkits are needed to assess the safety and efficacy of these new therapies and accelerate progress. Emerging technologies to monitor the biological effects of genome editors cover a range of biological scales, from the direct measurement of editing outcomes in DNA, to human microphysiological systems, and non-invasive in vivo imaging. Measurements of on-target and off-target editing outcomes, including sequences unique to humans, provide essential benchmarks to understand functional responses. Microphysiological systems, including organoids and organs-on-chips, enable phenotypic evaluations of editing strategies in varied organ lineages and disease states. Non-invasive imaging modalities can track the biodistribution and activities of genome editors and edited cells in vivo. Collectively, these technologies provide complementary insights across different scales, from the single nucleotide to the whole organism, bridging preclinical therapeutics development with clinical trials.

Indexed as

CRISPR-Cas SystemsGene EditingGenetic TherapyAnimalsHumansMicrophysiological Systems

Identifiers

PMID41530266
PMCPMC13101755

What OpenQuestion holds

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Registered trials

None linked

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.