ArticleMatters2018
Lentiviral gene therapy vector with UCOE stably restores function in iPSC-derived neutrophils of a CDG patient.
Open access · greenAbstract read
In one paragraph
Article in Matters, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
0.7field-weighted citation impact, top 24% 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
5 citing papers in PubMed, 7 citations in OpenAlex.
- CAR-engineered neutrophils derived from induced pluripotent stem cells: a new frontier in cellular immunotherapy.Clinical & translational oncology : official publication of the Federation of Spanish Oncology Societies and of the National Cancer Institute of Mexico · 2026Review
- Minimal CBX3-Derived UCOE Confers Long-Term Resistance to Transgene Silencing in Human iPSCs During Neuronal Differentiation.International journal of molecular sciences · 2026Article
- Genetic counseling for congenital disorders of glycosylation (CDG).Journal of genetic counseling · 2024Article
- Treatment Options in Congenital Disorders of Glycosylation.Frontiers in genetics · 2021Review
- Concise Review: Modeling Neurodegenerative Diseases with Human Pluripotent Stem Cell-Derived Microglia.Stem cells (Dayton, Ohio) · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 5 institutions in 3 countries.
Walther HaenselerSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Elena KuzmenkoSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Adjoa Smalls-ManteySystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Cathy BrowneSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Reinhard SegerSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
William S JamesSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Sally A CowleySystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Janine ReichenbachSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Ulrich SilerSystems and Cell Biology of Neurodegeneration, University of Zurich, James Martin Stem Cell Facility, University of Oxford, Division of Immunology, University Children's Hospital Zurich, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, Department of Psychiatry, Sir William Dunn School of Pathology, University of Oxford, Icahn School of Medicine at Mount Sinai, HIV-Specific Immunity Section, Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA, James Martin Stem Cell Facility, Sir William Dunn School of Pathology, University of Oxford, Professor emeritus University Children's Hospital Zürich, Division of Immunology, Professor emeritus Children's Research Center, Div. of Immunology, University Children's Hospital Zurich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich, Center for Applied Biotechnology and Molecular Medicine, University of Zürich, Div. of Immunology, University Children's Hospital Zürich, Children's Research Center, Associated Group Institute for Regenerative Medicine, University of Zürich.
Institute for Stem Cell Biology and Regenerative Medicine · INAssociated Research (United States) · USUniversity of Zurich · CHIcahn School of Medicine at Mount Sinai · USUniversity Children's Hospital Zurich · CH
Funding
Characterization of Antibody-Dependent Cellular Cytotoxicity in HIV InfectionF31AI098409 · NIAID · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI SMALLS-MANTEY, ADJOA R · 2012 to 2015
$121kNIAID NIH HHS F31 AI098409
Abstract
A recent gamma-retroviral clinical Chronic Granulomatous Disease (CGD) gene therapy (GT) trial achieved proof-of-concept but was accompanied by activation of oncogenes and transgene silencing. The
Indexed as
CGDClinical ImmunologyGenetherapyHematologyImmunologyIPSCNeutrophilsSilencing
Identifiers
What OpenQuestion holds
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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.