Evidence map›Paper›PMID 32000541›Full record

ArticleHuman gene therapy2020

High-Throughput

Adrian Westhaus, Marti Cabanes-Creus, Arkadiusz Rybicki, Grober Baltazar, Renina Gale Navarro, Erhua Zhu, Matthieu Drouyer, Maddison Knight, Razvan F Albu, Boaz H Ng and 10 more

Open access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 70 papers.

0numbers the graph read from it
0cells of the map it votes in
70citing papers in PubMed
10.8field-weighted citation impact, top 1% 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

70 citing papers in PubMed, 96 citations in OpenAlex.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Article
  7. Article
  8. Article
  9. Review
  10. Article
  11. Article
  12. Review
  13. Identification of a robust promoter in mouse and human hepatocytes by in vivo biopanning of a barcoded AAV library.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  14. Article
  15. Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  16. Article
  17. Article
  18. Article
  19. Article
  20. Review

10 more citing papers are in PubMed but not listed here.

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

20 authors at 4 institutions in 3 countries.

Adrian WesthausTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Marti Cabanes-CreusTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Arkadiusz RybickiTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Grober BaltazarTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Renina Gale NavarroTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Erhua ZhuGene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, The University of Sydney, Westmead, Australia.
Matthieu DrouyerTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
Maddison KnightVector and Genome Engineering Facility, Children's Medical Research Institute, , The University of Sydney, Westmead, Australia.
Razvan F AlbuVector and Genome Engineering Facility, Children's Medical Research Institute, , The University of Sydney, Westmead, Australia.
Boaz H NgVector and Genome Engineering Facility, Children's Medical Research Institute, , The University of Sydney, Westmead, Australia.
Predrag KalajdzicVector and Genome Engineering Facility, Children's Medical Research Institute, , The University of Sydney, Westmead, Australia.
Magdalena KwiatekMilitary Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, Puławy, Poland.
Kenneth HsuChildren's Cancer Research Unit, The Children's Hospital at Westmead, Westmead, Australia.
Giorgia SantilliGreat Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Wendy GoldMolecular Neurobiology Research Lab, The Children's Hospital at Westmead, Westmead, Australia.
Belinda KramerChildren's Cancer Research Unit, The Children's Hospital at Westmead, Westmead, Australia.
Anai Gonzalez-CorderoStem Cell & Organoid Facility and Stem Cell Medicine Group, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Adrian J ThrasherGreat Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, The University of Sydney, Westmead, Australia.
Leszek LisowskiTranslational Vectorology Research Unit, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.
The University of Sydney · AUChildren's Hospital at Westmead · AUGreat Ormond Street Hospital · GBWojskowy Instytut Higieny i Epidemiologii · PL

Funding

Department of HealthWellcome Trust 104807/Z/14/Z
6 · The paper itself

Abstract

Adeno-associated virus (AAV) vectors are quickly becoming the vectors of choice for therapeutic gene delivery. To date, hundreds of natural isolates and bioengineered variants have been reported. While factors such as high production titer and low immunoreactivity are important to consider, the ability to deliver the genetic payload (physical transduction) and to drive high transgene expression (functional transduction) remains the most important feature when selecting AAV variants for clinical applications. Reporter expression assays are the most commonly used methods for determining vector fitness. However, such approaches are time consuming and become impractical when evaluating a large number of variants. Limited access to primary human tissues or challenging model systems further complicates vector testing. To address this problem, convenient high-throughput methods based on next-generation sequencing (NGS) are being developed. To this end, we built an AAV Testing Kit that allows inherent flexibility in regard to number and type of AAV variants included, and is compatible with

Indexed as

AnimalsCapsidCell LineCell Line, TumorCells, CulturedDependovirusDNA, ViralFemaleFibroblastsGenetic TherapyGenetic VectorsGene Transfer TechniquesHeLa CellsHigh-Throughput Nucleotide SequencingHigh-Throughput Screening AssaysHumansDNA, ViralEPHB2 protein, humanReceptor, EphB2AAVadeno-associated virusgene therapyiPSCnext-generation sequencingviral vectors

Identifiers

PMID32000541
PMCPMC7232709
OpenAlexW3003692123

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
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.