ArticleScience translational medicine2024
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.
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Article in Science translational medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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13citing papers in PubMed
–field-weighted citation impact
What it found
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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
13 citing papers in PubMed.
- NADPH oxidases in immunometabolism and disease pathology: mechanistic networks, pollutant triggers, and therapeutic frontiers.Cellular & molecular immunology · 2026Review
- Gene editing of hematopoietic stem cells: applications and advances.International journal of hematology · 2026Review
- How I Treat: Chronic granulomatous disease.Journal of human immunity · 2026Review
- Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.Nature biomedical engineering · 2026Article
- Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.International journal of molecular sciences · 2026Review
- Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE.Nature biotechnology · 2026Article
- Review
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- Gene therapy for inborn errors of immunity: past progress, current status and future directions.Rare disease and orphan drugs journal · 2025Article
- Review
- Advancing gene editing therapeutics: Clinical trials and innovative delivery systems across diverse diseases.Molecular therapy. Nucleic acids · 2025Review
- Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- CRISPR/Cas9 Delivery Systems to Enhance Gene Editing Efficiency.International journal of molecular sciences · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
20 authors.
Vera BzhilyanskayaGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0002-7886-4613
Linyuan MaCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.ORCID 0000-0003-1097-1292
Siyuan LiuMolecular Cytogenetic Core Facility, Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Frederick, MD 21702, USA.
Lauren R FoxCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.ORCID 0009-0003-4828-8365
Madelynn N WhittakerCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.ORCID 0000-0002-3382-1134
Ronald J MeisCELLSCRIPT, Madison, WI 53713, USA.ORCID 0000-0001-9522-3036
Uimook ChoiGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
Amanda LawsonGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0009-0005-0269-0564
Michelle MaGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0009-0009-1742-7637
Narda TheobaldGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0001-6872-7925
Sandra BurkettMolecular Cytogenetic Core Facility, Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Frederick, MD 21702, USA.ORCID 0000-0002-5476-4028
Colin L SweeneyGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0002-9442-1134
Cicera R LazzarottoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0003-1093-0175
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.ORCID 0000-0001-9161-3993
Justin B LackBioinformatics (NCBR)/Integrated Data Sciences Section (IDSS), Research Technology Branch/DIR/NIAID, Frederick, MD 21702, USA.
Xiaolin WuMolecular Cytogenetic Core Facility, Mouse Cancer Genetics Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Frederick, MD 21702, USA.ORCID 0000-0002-6432-1300
Gary A DahlCELLSCRIPT, Madison, WI 53713, USA.
Harry L MalechGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0001-5874-5775
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.ORCID 0000-0002-5469-0655
Suk See De RavinGenetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD 20892, USA.ORCID 0000-0002-9800-774X
Funding
WORK ORDER 126643 B539 EXPAND IC SUITE75N91019D00024 · NIAID · LEIDOS BIOMEDICAL RESEARCH, INC. · PI BRISCOE, LYNN · 2019 to 2025
$3932.6MPROJECT 4: Somatic evolution of the hematopoietic system in cardiovascular diseaseP01HL142494 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Filip K Swirski · 2019 to 2026
$19.5MPERIPHERAL BLOOD PROGENITORS-TARGET FOR GENE TRANSFERZ01AI000644 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 1992 to 2008
$4.3MScalable Development of Custom Genome Editing TechnologiesDP2CA281401 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2022 to 2025
$2.5MUltra-sensitive, unbiased, high-throughput, biochemical CHANGE-seq genome-wide activity and gRNA sequencing assays for therapeutic genome editing INDsU01AI176471 · NIAID · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI TSAI, SHENGDAR · 2023 to 2025
$1.4MGENE THERAPY FOR IMMUNE DEFICIENCIESZ01AI000645 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI DE RAVIN, SUK · 1992 to 2008
$873kBasic Research on Hematopoietic Adult Human Stem CellsZ01AI000988 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 2007 to 2008
$669kIntramural NIH HHS Z01 AI000644Intramural NIH HHS Z01 AI000645Intramural NIH HHS Z01 AI000988NCI NIH HHS 75N91019D00024NCI NIH HHS DP2 CA281401NHLBI NIH HHS P01 HL142494NIAID NIH HHS U01 AI176471
Abstract
X-linked chronic granulomatous disease (X-CGD) is an inborn error of immunity (IEI) resulting from genetic mutations in the cytochrome b-245 beta chain (
Indexed as
Gene EditingGranulomatous Disease, ChronicHematopoietic Stem CellsMutationAnimalsCRISPR-Cas SystemsHematopoietic Stem Cell TransplantationHumansMiceNADPH Oxidase 2NADPH Oxidase 2
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