ArticleHuman gene therapy2023
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Erratum issuedOpen access · hybridAbstract read
In one paragraph
Article in Human gene therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 14 papers.
0numbers the graph read from it
0cells of the map it votes in
14citing papers in PubMed
7.2field-weighted citation impact, top 3% 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed, 23 citations in OpenAlex.
- Defining the safety and efficacy of liver-directed AAV gene therapy using a human liver tissue-equivalent platform.Molecular therapy. Advances · 2026Article
- Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression.Therapeutic advances in hematology · 2026Review
- Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.Molecular therapy. Methods & clinical development · 2025Article
- AAV capsid prioritization in normal and steatotic human livers maintained by machine perfusion.Nature biotechnology · 2025Article
- Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Article
- Article
- Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.Nature communications · 2024Article
- Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- A humanized mouse model for adeno-associated viral gene therapy.Nature communications · 2024Article
- Recent advances in various adeno-associated viruses (AAVs) as gene therapy agents in hepatocellular carcinoma.Virology journal · 2024Review
- Miniaturization of hiPSC-derived 3D neural cultures in stirred-tank bioreactors for parallelized preclinical assessment of rAAV.Frontiers in bioengineering and biotechnology · 2024Article
- AAV capsid bioengineering in primary human retina models.Scientific reports · 2023Article
Corrections and comments
- Erratum issued
Authors and funding
27 authors at 10 institutions in 5 countries.
Adrian WesthausTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.ORCID 0000-0001-6439-4326
Marti Cabanes-CreusTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Kimberley L DilworthTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Erhua ZhuGene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
David Salas GómezGene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain.
Renina G NavarroTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Anais K AmayaGene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.ORCID 0000-0002-8396-2226
Suzanne ScottTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Magdalena KwiatekMilitary Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, Puławy, Poland.
Alexandra L McCorkindaleInventia Life Science Pty Ltd., Sydney, Australia.
Tara E HaymanInventia Life Science Pty Ltd., Sydney, Australia.
Silke FrahmStem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany.
Dany P PerocheauGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Bang Manh TranDepartment of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Elizabeth VincanDepartment of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Sharon L WongMolecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.
Shafagh A WatersMolecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.
Georgina E RiddioughDepartment of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Marcos V PeriniDepartment of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia.
Laurence O W WilsonAustralian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia.
Julien BaruteauGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Sebastian DieckeStem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany.
Gloria González-AseguinolazaGene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain.ORCID 0000-0002-1600-4562
Giorgia SantilliGreat Ormond Street Institute of Child Health, University College London, London, United Kingdom.ORCID 0000-0003-1776-1984
Adrian J ThrasherGreat Ormond Street Institute of Child Health, University College London, London, United Kingdom.ORCID 0000-0002-6097-6115
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Leszek LisowskiTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.ORCID 0000-0002-4772-8651
The University of Sydney · AUGreat Ormond Street Hospital · GBCommonwealth Scientific and Industrial Research Organisation · AUMax Delbrück Center · DENavarre Institute of Health Research · ESThe University of Melbourne · AUUNSW Sydney · AUAustin Health · AUThe Royal Melbourne Hospital · AUWojskowy Instytut Higieny i Epidemiologii · PL
Funding
Department of HealthMedical Research Council MR/T008024/1Wellcome Trust 217112/Z/19/Z
Abstract
The liver is a prime target for
Indexed as
DependovirusLiverCapsid ProteinsGenetic TherapyGenetic VectorsHepatocytesHumansTropismCapsid ProteinsAAVhepatocyteliver gene therapynonhuman primatexenograft model
Identifiers
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.