Evidence map›Paper›PMID 38438373›Full record

ArticleNature communications2024

A humanized mouse model for adeno-associated viral gene therapy.

Mercedes Barzi, Tong Chen, Trevor J Gonzalez, Francis P Pankowicz, Seh Hoon Oh, Helen L Streff, Alan Rosales, Yunhan Ma, Sabrina Collias, Sarah E Woodfield and 8 more

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
5.5field-weighted citation impact, top 4% 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

10 citing papers in PubMed, 10 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Article
  5. Article
  6. AAVR Expression is Essential for AAV Vector Transduction in Sensory Hair Cells.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Article
  7. A comprehensive atlas of AAV tropism in the mouse.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  8. Article
  9. Mice Engrafted with Human Liver Cells.Seminars in liver disease · 2024
    Review
  10. 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

18 authors at 4 institutions in 1 country.

Mercedes Barzi *Alice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA.
Tong Chen *Alice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA.
Trevor J GonzalezDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, 27710, USA.
Francis P PankowiczCenter for Cell and Gene Therapy, Stem Cells and Regenerative Medicine Center, Baylor College of Medicine, Houston, TX, 77030, USA.
Seh Hoon OhDepartment of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC, 27710, USA.
Helen L StreffDepartment of Biomedical Engineering, Duke University Pratt School of Engineering, Duke University, Durham, NC, USA.ORCID 0000-0003-3582-3923
Alan RosalesDepartment of Biomedical Engineering, Duke University Pratt School of Engineering, Duke University, Durham, NC, USA.ORCID 0000-0001-6986-2985
Yunhan MaAlice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA.
Sabrina ColliasAlice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA.
Sarah E WoodfieldMichael E. DeBakey Department of Surgery, Divisions of Pediatric Surgery and Surgical Research, Baylor College of Medicine, Houston, TX, 77030, USA.
Anna Mae DiehlDepartment of Medicine, Division of Gastroenterology, Duke University Medical Center, Durham, NC, 27710, USA.ORCID 0000-0003-1859-089X
Sanjeev A VasudevanMichael E. DeBakey Department of Surgery, Divisions of Pediatric Surgery and Surgical Research, Baylor College of Medicine, Houston, TX, 77030, USA.
Thao N GalvanDepartment of Surgery, Texas Children's Hospital, Houston, TX, 77030, USA.ORCID 0000-0002-1289-4692
John GossDepartment of Surgery, Texas Children's Hospital, Houston, TX, 77030, USA.
Charles A GersbachDepartment of Biomedical Engineering, Duke University Pratt School of Engineering, Duke University, Durham, NC, USA.ORCID 0000-0003-1478-4013
Beatrice Bissig-ChoisatAlice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA.
Aravind AsokanDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, 27710, USA.ORCID 0000-0001-5563-4877
Karl-Dimiter BissigAlice and Y. T. Chen Center for Genetics and Genomics, Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, 27710, USA. karldimiter.bissig@duke.edu.ORCID 0000-0002-9577-9250
Duke Medical Center · USDuke University · USBaylor College of Medicine · USMichael E. DeBakey VA Medical Center · US

Funding

Dissecting AAV silencing in humanized miceR01DK134408 · NIDDK · DUKE UNIVERSITY · PI Aravind Asokan, Karl-Dimiter Bissig · 2023 to 2026
$2.4M
Microphysiological Human Tissue Systems for Monitoring of Genome Editing OutcomesU01EB028901 · NIBIB · DUKE UNIVERSITY · PI BURSAC, NENAD, GERSBACH, CHARLES A. · 2019 to 2019
$668k
NIBIB NIH HHS U01 EB028901NIDDK NIH HHS R01 DK134408
6 · The paper itself

Abstract

Clinical translation of AAV-mediated gene therapy requires preclinical development across different experimental models, often confounded by variable transduction efficiency. Here, we describe a human liver chimeric transgene-free Il2rg

Indexed as

DependovirusLiverAnimalsDisease Models, AnimalGenetic TherapyHepatocytesHumansMice

Identifiers

PMID38438373
PMCPMC10912671
OpenAlexW4392376488

What OpenQuestion holds

Textmetadata
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.