ArticleHuman gene therapy2017
Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.
Registry-linked trialOpen access · greenAbstract read
In one paragraph
Article in Human gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03538899 (Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency), which is not on this map. Cited by 26 papers.
0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
3.9field-weighted citation impact, top 7% 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.
NCT03538899 phase1 / phase2recruitingnot on this mapstarted 2018, after this paper: background citation
Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells
Who cites it
26 citing papers in PubMed, 53 citations in OpenAlex.
- Lentiviral Gene Therapy for Artemis-Deficient SCID.The New England journal of medicine · 2022Trial
- Base editing of Artemis mutations ex vivo sheds light on gene therapy for Artemis-deficient SCID.Advanced biotechnology · 2026Article
- Leucine zipper-based immunomagnetic purification of CAR T cells displaying multiple receptors.Nature biomedical engineering · 2024Article
- Severe Combined Immunodeficiency from a Homozygous DNA Ligase 1 Mutant with Reduced Catalytic Activity but Increased Ligation Fidelity.Journal of clinical immunology · 2024Article
- Development of anMolecular therapy. Methods & clinical development · 2023Article
- Clinical and Genetic Characterization of Patients with Artemis Deficiency in Japan.Journal of clinical immunology · 2023Article
- Correcting inborn errors of immunity: From viral mediated gene addition to gene editing.Seminars in immunology · 2023Review
- Precise somatic genome editing for treatment of inborn errors of immunity.Frontiers in immunology · 2022Review
- Precision medicine: The use of tailored therapy in primary immunodeficiencies.Frontiers in immunology · 2022Review
- The Role of DNA Repair in Immunological Diversity: From Molecular Mechanisms to Clinical Ramifications.Frontiers in immunology · 2022Review
- Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Gene Therapy for Primary Immunodeficiency.HemaSphere · 2021Review
- Update on DNA-Double Strand Break Repair Defects in Combined Primary Immunodeficiency.Current allergy and asthma reports · 2020Review
- Induced Pluripotent Stem Cell Meets Severe Combined Immunodeficiency.Cell journal · 2020Review
- Preclinical Development of Autologous Hematopoietic Stem Cell-Based Gene Therapy for Immune Deficiencies: A Journey from Mouse Cage to Bed Side.Pharmaceutics · 2020Review
- Gene therapy for severe combined immunodeficiencies and beyond.The Journal of experimental medicine · 2020Review
- A Novel Branched DNA-Based Flowcytometric Method for Single-Cell Characterization of Gene Therapy Products and Expression of Therapeutic Genes.Frontiers in immunology · 2020Article
- Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID.Molecular therapy. Methods & clinical development · 2019Article
- Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.Nature reviews. Drug discovery · 2019Review
- Newborn screening for severe combined immunodeficiency and T-cell lymphopenia.Immunological reviews · 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
17 authors at 6 institutions in 2 countries.
Divya Punwani1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Misako Kawahara1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Jason Yu1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Ukina Sanford1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Sushmita Roy1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Kiran Patel1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Denise A Carbonaro2 Departments of Microbiology, Immunology and Molecular Genetics and Pediatrics, University of California Los Angeles , Los Angeles, California.
Andrea D Karlen3 Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis, Minnesota.
Sara Khan1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Kenneth Cornetta4 Department of Medical and Molecular Genetics, Indiana University, and the Indiana University Viral Production Facility, Indianapolis, Indiana.
Michael Rothe5 Institute for Experimental Hematology, Hannover Medical School , Hannover, Germany.
Axel Schambach5 Institute for Experimental Hematology, Hannover Medical School , Hannover, Germany.
Donald B Kohn2 Departments of Microbiology, Immunology and Molecular Genetics and Pediatrics, University of California Los Angeles , Los Angeles, California.
Harry L Malech6 Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, Division of Intramural Research, National Institutes of Health , Bethesda, Maryland.
R Scott McIvor3 Department of Genetics, Cell Biology and Development, University of Minnesota , Minneapolis, Minnesota.
Jennifer M Puck1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
Morton J Cowan1 Department of Pediatrics, University of California School of Medicine and University of California San Francisco Benioff Children's Hospital , San Francisco, San Francisco, California.
University of California, San Francisco · USMedizinische Hochschule Hannover · DEUniversity of California, Los Angeles · USUniversity of Minnesota · USIndiana University – Purdue University Indianapolis · USNational Institutes of Health · US
Funding
Training of Clinical Investigators in Primary Immune DeficienciesU54AI082973 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI DVORAK, CHRISTOPHER CRAIG, HADDAD, ELIE · 2009 to 2023
$23.3MGene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1MBasic Research on Hematopoietic Human Stem CellsZIAAI000988 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2019
$6.5MGene Therapy Clinical Trials for X-linked Severe Combined ImmunodeficiencyZIAAI000992 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK · 2009 to 2019
$4.1MNewborn Screening for T-Cell Disorders: Spectrum and OutcomesR01AI105776 · NIAID · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI BRENNER, STEVEN E, CHURCH, JOSEPH · 2013 to 2016
$2.4MNewborn Screening for SCID in a High-Risk PopulationR03HD060311 · NICHD · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI PUCK, JENNIFER M. · 2008 to 2009
$155kNIAID NIH HHS R01 AI105776NIAID NIH HHS U54 AI082973NICHD NIH HHS R03 HD060311
Abstract
During B and T lymphocyte maturation, V(D)J recombination is initiated by creation of DNA double-strand breaks. Artemis is an exonuclease essential for their subsequent repair by nonhomologous end-joining. Mutations in DCLRE1C, the gene encoding Artemis, cause T
Indexed as
Genetic TherapyAnimalsB-LymphocytesCells, CulturedCombined Modality TherapyDisease Models, AnimalDNA-Binding ProteinsDNA RepairEndonucleasesFibroblastsGamma RaysGenetic VectorsHematopoietic Stem CellsHematopoietic Stem Cell TransplantationHumansLentivirusDCLRE1C protein, humanDclre1c protein, mouseDNA-Binding ProteinsEndonucleasesNuclear ProteinsArtemisgene therapylentivirusradiation sensitivitysevere combined immunodeficiency
Identifiers
What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
NCT03538899ClinicalTrials.gov
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.