Evidence map›Paper›PMID 31513879›Full record

ArticleThe Journal of allergy and clinical immunology2020

Intrathymic adeno-associated virus gene transfer rapidly restores thymic function and long-term persistence of gene-corrected T cells.

Marie Pouzolles, Alice Machado, Mickaël Guilbaud, Magali Irla, Sarah Gailhac, Pierre Barennes, Daniela Cesana, Andrea Calabria, Fabrizio Benedicenti, Arnauld Sergé and 7 more

Open access · bronzeAbstract read
In one paragraph

Article in The Journal of allergy and clinical immunology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
1.1field-weighted citation impact, top 20% of its field
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

8 citing papers in PubMed, 14 citations in OpenAlex.

  1. Review
  2. Article
  3. In vivo CAR cell therapy: from bench to bedside.Journal of hematology & oncology · 2025
    Review
  4. T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  5. Delivering CRISPR to the HIV-1 reservoirs.Frontiers in microbiology · 2024
    Review
  6. Article
  7. AAV integration in human hepatocytes.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Article
  8. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

17 authors at 4 institutions in 3 countries.

Marie PouzollesInstitut de Génétique Moléculaire de Montpellier, University of Montpellier, CNRS, Montpellier, France.
Alice MachadoInstitut de Génétique Moléculaire de Montpellier, University of Montpellier, CNRS, Montpellier, France.
Mickaël GuilbaudINSERM UMR1089, Université de Nantes, Centre Hospitalier Universitaire de Nantes, Nantes, France.
Magali IrlaCenter of Immunology Marseille-Luminy (CIML), INSERM U1104, CNRS UMR7280, Aix-Marseille Université UM2, Marseille, France.
Sarah GailhacInstitut de Génétique Moléculaire de Montpellier, University of Montpellier, CNRS, Montpellier, France.
Pierre BarennesSorbonne Université, INSERM, Immunology-Immunopathology-Immunotherapy (i3), Paris, France.
Daniela CesanaSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS, San Raffaele Scientific Institute, Milan, Italy.
Andrea CalabriaSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS, San Raffaele Scientific Institute, Milan, Italy.
Fabrizio BenedicentiSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS, San Raffaele Scientific Institute, Milan, Italy.
Arnauld SergéAix Marseille University, CNRS, INSERM, Institut Paoli-Calmettes, CRCM, Marseille, France.
Indu RamanMicroarray Core Facility, University of Texas Southwestern Medical Center, Dallas, Tex.
Quan-Zhen LiMicroarray Core Facility, University of Texas Southwestern Medical Center, Dallas, Tex; Department of Immunology, University of Texas Southwestern Medical Center, Dallas, Tex.
Eugenio MontiniSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS, San Raffaele Scientific Institute, Milan, Italy.
David KlatzmannSorbonne Université, INSERM, Immunology-Immunopathology-Immunotherapy (i3), Paris, France; AP-HP, Hôpital Pitié-Salpêtrière, Biotherapy (CIC-BTi) and Inflammation-Immunopathology-Biotherapy Department (i2B), Paris, France.
Oumeya AdjaliINSERM UMR1089, Université de Nantes, Centre Hospitalier Universitaire de Nantes, Nantes, France. Electronic address: oumeya.adjali@univ-nantes.fr.
Naomi TaylorInstitut de Génétique Moléculaire de Montpellier, University of Montpellier, CNRS, Montpellier, France; Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Md. Electronic address: taylorn4@mail.nih.gov.
Valérie S ZimmermannInstitut de Génétique Moléculaire de Montpellier, University of Montpellier, CNRS, Montpellier, France. Electronic address: zimmermann@igmm.cnrs.fr.
Centre National de la Recherche Scientifique · FRInserm · FRIRCCS Ospedale San Raffaele · ITThe University of Texas Southwestern Medical Center · US

Funding

Reprogramming hematopoietic stem cell (HSC) differentiation via metabolic conditZIABC011924 · NCI · DIVISION OF BASIC SCIENCES - NCI · PI TAYLOR, NAOMI · 2019 to 2025
$9.0M
ZAP-70 Deficiency: A model for gene therapy studiesR01AI059349 · NIAID · CNRS DELEGATION LANGUEDOC-ROUSSILLON · PI TAYLOR, NAOMI · 2007 to 2010
$812k
Intramural NIH HHS ZIA BC011924NIAID NIH HHS R01 AI059349
6 · The paper itself

Abstract

backgroundPatients with T-cell immunodeficiencies are generally treated with allogeneic hematopoietic stem cell transplantation, but alternatives are needed for patients without matched donors. An innovative intrathymic gene therapy approach that directly targets the thymus might improve outcomes.

objectiveWe sought to determine the efficacy of intrathymic adeno-associated virus (AAV) serotypes to transduce thymocyte subsets and correct the T-cell immunodeficiency in a zeta-associated protein of 70 kDa (ZAP-70)-deficient murine model.

methodsAAV serotypes were injected intrathymically into wild-type mice, and gene transfer efficiency was monitored. ZAP-70

resultsAAV8, AAV9, and AAV10 serotypes all transduced thymocyte subsets after in situ gene transfer, with transduction of up to 5% of cells. Intrathymic injection of an AAV8-ZAP-70 vector into ZAP-70

conclusionsIntrathymic AAV-transduced progenitors promote a rapid restoration of the thymic architecture, with a single wave of thymopoiesis generating long-term peripheral T-cell function.

Indexed as

ThymocytesZAP-70 Protein-Tyrosine KinaseAnimalsDependovirusGenetic TherapyGenetic VectorsImmunologic Deficiency SyndromesMiceMice, KnockoutTransduction, GeneticZAP-70 Protein-Tyrosine Kinasegene therapyhumoral immunityintrathymic gene transfermedulla formationSevere combined immunodeficiencyT-cell reconstitutionthymuszeta-associated protein of 70kDa

Identifiers

PMID31513879
PMCPMC8287836
OpenAlexW2973028058

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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.