Evidence map›Paper›PMID 40744920›Full record

ArticleNature communications2025

GFP-on mouse model for interrogation of in vivo gene editing.

Carla Dib, Jack A Queenan, Leah Swartzrock, Hana Willner, Morgane Denis, Nouraiz Ahmed, Fareha Moulana Zada, Beltran Borges, Carsten T Charlesworth, Tony Lum and 12 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Nature communications, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  2. Antigen-directed p53 restoration inFrontiers in oncology · 2026
    Article
  3. A fluorescent reporter system for trackingFrontiers in microbiology · 2026
    Article
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

22 authors.

Carla Dib *Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Jack A Queenan *Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-1057-3892
Leah SwartzrockDepartment of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0003-0584-0896
Hana WillnerDepartment of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Morgane DenisDepartment of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-9485-0131
Nouraiz AhmedMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Fareha Moulana ZadaDepartment of Surgery, University of California San Francisco, San Francisco, CA, USA.ORCID http://orcid.org/0009-0007-6676-3384
Beltran BorgesDepartment of Surgery, University of California San Francisco, San Francisco, CA, USA.
Carsten T CharlesworthInstitute for Stem Cell Biology and Regenerative Medicine, Stanford University, Stanford, CA, USA.
Tony LumDepartment of Surgery, University of California San Francisco, San Francisco, CA, USA.
Bradley P YatesDepartment of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Caleb Y KwonDartmouth College, Thayer School of Engineering, Hanover, NH, USA.
Augustino V ScorzoDartmouth College, Thayer School of Engineering, Hanover, NH, USA.ORCID http://orcid.org/0000-0001-5573-5985
Scott C DavisDartmouth College, Thayer School of Engineering, Hanover, NH, USA.
Jessie R DavisMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Ran HeDepartment of Genetics & Cellular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, USA.
Jun XieDepartment of Genetics & Cellular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0001-9565-1567
Guangping GaoDepartment of Genetics & Cellular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, USA.ORCID http://orcid.org/0000-0003-0097-9012
Tippi C MacKenzieDepartment of Surgery, University of California San Francisco, San Francisco, CA, USA.
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA. drliu@fas.harvard.edu.ORCID http://orcid.org/0000-0002-9943-7557
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA. gnewby@jhmi.edu.ORCID http://orcid.org/0000-0001-7869-2615
Agnieszka D CzechowiczDepartment of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA. aneeshka@stanford.edu.ORCID http://orcid.org/0000-0003-2909-2669

Funding

Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
Delivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3M
Expanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1M
In vivo precision genome editing to correct genetic diseaseR00HL163805 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEWBY, GREGORY A. · 2023 to 2025
$747k
In vivo precision genome editing to correct genetic diseaseK99HL163805 · NHLBI · BROAD INSTITUTE, INC. · PI NEWBY, GREGORY A. · 2022 to 2022
$167k
NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS K99 HL163805NHLBI NIH HHS R00 HL163805NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIGMS NIH HHS R35 GM118062
6 · The paper itself

Abstract

Gene editing technologies have revolutionized therapies for numerous genetic diseases. However, in vivo gene editing hinges on identifying efficient delivery vehicles for editing in targeted cell types, a significant hurdle in fully realizing its therapeutic potential. A model system to rapidly evaluate systemic gene editing would advance the field. Here, we develop the GFP-on reporter mouse, which harbors a nonsense mutation in a genomic EGFP sequence correctable by adenine base editor (ABE) among other genome editors. The GFP-on system was validated using single and dual adeno-associated virus (AAV9) encoding ABE8e and sgRNA. Intravenous administration of AAV9-ABE8e-sgRNA into adult GFP-on mice results in EGFP expression consistent with the tropism of AAV9. Intrahepatic delivery of AAV9-ABE8e-sgRNA into GFP-on fetal mice restores EGFP expression in AAV9-targeted organs lasting at least six months post-treatment. The GFP-on model provides an ideal platform for high-throughput evaluation of emerging gene editing tools and delivery modalities.

Indexed as

Gene EditingGreen Fluorescent ProteinsAnimalsCodon, NonsenseCRISPR-Cas SystemsDependovirusFemaleGenes, ReporterGenetic VectorsHumansMiceRNA, Guide, CRISPR-Cas SystemsCodon, Nonsenseenhanced green fluorescent proteinGreen Fluorescent ProteinsRNA, Guide, CRISPR-Cas Systems

Identifiers

PMID40744920
PMCPMC12313916

What OpenQuestion holds

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Registered trials

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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.