Evidence map›Paper›PMID 39863928›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

A comprehensive atlas of AAV tropism in the mouse.

Christopher J Walkey, Kathy J Snow, Jote Bulcha, Aaron R Cox, Alexa E Martinez, M Cecilia Ljungberg, Denise G Lanza, Marco De Giorgi, Marcel A Chuecos, Michele Alves-Bezerra and 16 more

Abstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 34 papers.

0numbers the graph read from it
0cells of the map it votes in
34citing papers in PubMed
–field-weighted citation impact
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

34 citing papers in PubMed.

  1. Review
  2. Engineered Transformer Base Editor with Enhanced Editing Efficiency.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Article
  3. Article
  4. Article
  5. Review
  6. Article
  7. AAV9-MediatedPharmaceutics · 2026
    Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Review
  17. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  18. Article
  19. Article
  20. Past, Present and Future of Regenerative Gene Therapy for Ischemic Heart Failure.Journal of cardiovascular translational research · 2026
    Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

26 authors.

Christopher J WalkeyDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Kathy J SnowThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Jote BulchaHorae Gene Therapy Center and Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Aaron R CoxSection of Diabetes, Endocrinology, and Metabolism, Department of Medicine, Baylor College of Medicine, Houston, TX 77030, USA.
Alexa E MartinezDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
M Cecilia LjungbergDepartment of Pediatrics, Baylor College of Medicine, Houston, TX 77030, USA; Duncan Neurological Research Institute, Texas Children's Hospital, Houston, TX 77030, USA.
Denise G LanzaDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Marco De GiorgiDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Marcel A ChuecosDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Michele Alves-BezerraDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Carlos Flores SuarezDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Sean M HartigSection of Diabetes, Endocrinology, and Metabolism, Department of Medicine, Baylor College of Medicine, Houston, TX 77030, USA; Department of Molecular and Cellular Biology, Baylor College of Medicine, Houston, TX 77030, USA.
Susan G HilsenbeckDan L. Duncan Comprehensive Cancer Center, Baylor College of Medicine, Houston, TX, USA.
Chih-Wei HsuDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Ethan SavilleThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Yaned GaitanThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Jeff DuryeaThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Seth HanniganThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Mary E DickinsonDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA.
Oleg MirochnitchenkoOffice of Research Infrastructure Programs, Division of Program Coordination, Planning, and Strategic Initiatives, Office of the Director, National Institutes of Health, Bethesda, MD USA.
Dan WangHorae Gene Therapy Center and Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Cathleen M LutzThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
Jason D HeaneyDepartment of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Guangping GaoHorae Gene Therapy Center and Department of Microbiology and Physiological Systems, University of Massachusetts Medical School, Worcester, MA 01605, USA; Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA 01605, USA.
Stephen A MurrayThe Jackson Laboratory, 600 Main Street, Bar Harbor, ME 04609 USA.
William R LagorDepartment of Integrative Physiology, Baylor College of Medicine, Houston, TX 77030, USA. Electronic address: william.lagor@bcm.edu.

Funding

Shared Resource ManagementP30CA034196 · NCI · JACKSON LABORATORY · PI Paul Robson · 1985 to 2026
$61.9M
Genetic Repair of Familial HypercholesterolemiaR01HL132840 · NHLBI · BAYLOR COLLEGE OF MEDICINE · PI LAGOR, WILLIAM RAYMOND · 2017 to 2025
$5.4M
Physiological Dissection of the Mevalonate PathwayR01DK124477 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI William Raymond Lagor, NOAH Freeman SHROYER · 2020 to 2026
$3.5M
BCM-Rice resource for the analysis of somatic gene editing in miceU42OD026645 · OD · BAYLOR COLLEGE OF MEDICINE · PI DICKINSON, MARY E, HEANEY, JASON D. · 2018 to 2022
$3.5M
Resource SectionU42OD035581 · OD · BAYLOR COLLEGE OF MEDICINE · PI Jason D. Heaney, William Raymond Lagor · 2023 to 2026
$3.2M
Velcro AAV Vector for tissue-specific delivery of genome editing reagents with enhanced cargo capacityUG3HL151545 · NHLBI · RICE UNIVERSITY · PI BAO, GANG, LAGOR, WILLIAM RAYMOND · 2019 to 2021
$2.3M
Leptin regulation of delta cell functionR01DK136694 · NIDDK · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI RICHARD A COX · 2023 to 2026
$1.9M
LEPTIN REGULATION OF BETA CELL PROLIFERATIONR56DK128098 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI COX, RICHARD A · 2022 to 2022
$200k
Acyl-CoA synthetase-mediated regulation of lipid homeostasisK01DK128226 · NIDDK · BAYLOR COLLEGE OF MEDICINE · PI ALVES-BEZERRA, MICHELE · 2021 to 2021
$127k
NCI NIH HHS P30 CA034196NHLBI NIH HHS R01 HL132840NHLBI NIH HHS UG3 HL151545NIDDK NIH HHS K01 DK128226NIDDK NIH HHS R01 DK124477NIDDK NIH HHS R01 DK136694NIDDK NIH HHS R56 DK128098NIH HHS U42 OD026645NIH HHS U42 OD035581
6 · The paper itself

Abstract

Gene therapy with adeno-associated virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of 10 naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10, and AAVrh74) following systemic delivery into male and female mice. A transgene-expressing ZsGreen and Cre recombinase was used to identify transduction in a cell-dependent manner based on fluorescence. Cre-driven activation of tdTomato fluorescence offered superior sensitivity for transduced cells. All serotypes except AAV3B and AAV4 had high liver tropism. Fluorescence activation revealed transduction of unexpected tissues, including adrenals, testes, and ovaries. Rare transduced cells within tissues were also readily visualized. Biodistribution of AAV genomes correlated with fluorescence, except in immune tissues. AAV4 was found to have a pan-endothelial tropism while also targeting pancreatic beta cells. This public resource enables selection of the best AAV serotypes for basic science and preclinical applications in mice.

Indexed as

DependovirusGenetic VectorsViral TropismAnimalsFemaleGenetic TherapyHumansMaleMiceSerogroupTissue DistributionTransduction, GeneticTransgenesAAVAAV tropismadeno-associated virusbiodistributionfluorescent reportergene therapymouse modelsserotypestransduction

Identifiers

PMID39863928
PMCPMC11897767

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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.