Evidence map›Paper›PMID 40796771›Full record

ArticleNature communications2025

Targeted gene editing and near-universal cDNA insertion of CYBA and CYBB as a treatment for chronic granulomatous disease.

Jonas Holst Wolff, Thomas Wisbech Skov, Didde Haslund, Sofie Rahbek Dorset, Anne Louise S Revenfeld, Clotilde Aussel, Sofie E Jørgensen, Mette Holm, Martin K Thomsen, Sandra Ammann and 5 more

Abstract read
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 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. How I Treat: Chronic granulomatous disease.Journal of human immunity · 2026
    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

15 authors.

Jonas Holst WolffDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.ORCID http://orcid.org/0000-0001-5690-7603
Thomas Wisbech SkovDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.
Didde HaslundDepartment of Clinical Immunology, Aarhus University Hospital, Aarhus, Denmark.ORCID http://orcid.org/0000-0002-9890-5814
Sofie Rahbek DorsetDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.
Anne Louise S RevenfeldDepartment of Clinical Immunology, Aarhus University Hospital, Aarhus, Denmark.
Clotilde AusselInstitute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.
Sofie E JørgensenDepartment of Infectious Diseases, Aarhus University Hospital, Aarhus, Denmark.
Mette HolmDepartment of Paediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
Martin K ThomsenDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.ORCID http://orcid.org/0000-0002-5055-7531
Sandra AmmannInstitute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.ORCID http://orcid.org/0000-0003-0385-1890
Toni CathomenInstitute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, Freiburg, Germany.ORCID http://orcid.org/0000-0002-7757-4630
Trine H MogensenDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.ORCID http://orcid.org/0000-0002-1853-9704
Bjarne Kuno MøllerDepartment of Clinical Immunology, Aarhus University Hospital, Aarhus, Denmark.
Rasmus O BakDepartment of Biomedicine, Aarhus University, Aarhus, Denmark.ORCID http://orcid.org/0000-0002-7383-0297
Jacob Giehm MikkelsenDepartment of Biomedicine, Aarhus University, Aarhus, Denmark. giehm@biomed.au.dk.ORCID http://orcid.org/0000-0002-1322-3209

Funding

Carlsbergfondet (Carlsberg Foundation) CF21-0363Deutsche Forschungsgemeinschaft (German Research Foundation) CA 311/4-1 & FANEDIT/EJPRD20-209Innovationsfonden (Innovation Fund Denmark) 8056-00010ANovo Nordisk Fonden (Novo Nordisk Foundation) NNF220C0080684
6 · The paper itself

Abstract

Chronic granulomatous disease (CGD) is a severe inborn error of immunity caused by NADPH oxidase defects. Here, we develop CRISPR/Cas9-based gene editing strategies for correction of variants in the CYBA and CYBB genes causing CGD. For X-linked CGD, we also develop a near-universal gene editing strategy by targeted integration of a truncated CYBB cDNA in CD34

Indexed as

DNA, ComplementaryGene EditingGranulomatous Disease, ChronicNADPH Oxidase 2NADPH OxidasesAnimalsCRISPR-Cas SystemsGenetic TherapyHematopoietic Stem CellsHematopoietic Stem Cell TransplantationHumansMiceCYBB protein, humanDNA, ComplementaryNADPH Oxidase 2NADPH Oxidases

Identifiers

PMID40796771
PMCPMC12343970

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

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.