Evidence map›Paper›PMID 40683992›Full record

ArticleGene therapy2026

High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy.

Dhanya Ravindran, Renuka Rao, Juan Mundisugih, Tracy Titus, Shinya Tsurusaki, Cindy Y Kok, Fairooj N Rashid, Sindhu Igoor, Yasuhito Kotake, Saurabh Kumar and 4 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Review
  2. Article
  3. Review
  4. Review
  5. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

14 authors.

Dhanya Ravindran *Centre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.ORCID 0000-0002-9400-6088
Renuka Rao *Centre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Juan MundisugihCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Tracy TitusCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Shinya TsurusakiCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.ORCID 0000-0002-2592-4555
Cindy Y KokCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Fairooj N RashidCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Sindhu IgoorCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.
Yasuhito KotakeDepartment of Cardiology, Westmead Hospital, Westmead, NSW, Australia.
Saurabh KumarFaculty of Medicine and Health, The University of Sydney, Sydney, NSW, Australia.
James J H ChongCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia.ORCID 0000-0002-5201-4856
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute and Children's Hospital at Westmead, The University of Sydney and Sydney Children's Hospital Network, Westmead, NSW, Australia.ORCID 0000-0002-6213-5627
Leszek LisowskiFaculty of Medicine and Health, The University of Sydney, Sydney, NSW, Australia.ORCID 0000-0002-4772-8651
Eddy KizanaCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW, Australia. eddy.kizana@sydney.edu.au.ORCID 0000-0002-7266-6720

Funding

Department of Health | National Health and Medical Research Council (NHMRC) APP1184929Department of Health | National Health and Medical Research Council (NHMRC) APP1188348Department of Health | National Health and Medical Research Council (NHMRC) APP1194139
6 · The paper itself

Abstract

The selection of an appropriate promoter is important to the design and optimisation of adeno-associated viral (AAV) vector-based cardiac gene therapies. The expression cassette design can impact efficacy and safety of the vector. This study is the first to use a novel AAV barcode-seq method for the simultaneous evaluation of a panel of cardiac-specific promoters in a high-throughput manner. Functional analyses of our cardiac promoter kit packaged in three different capsids were performed using neonatal rat ventricular myocytes (NRVM), human iPSC-derived cardiomyocytes (hiPSC-CMs), HuH7 hepatocellular carcinoma cells, as well as mouse, rat, sheep and pig models. The cardiac troponin T (cTnT) promoter showed the most promise overall as a cardiac-specific promoter across all cardiac models tested. The results validate the barcode-seq technique as a powerful and versatile approach that enables high-throughput, quantitative analysis of various expression cassettes in commonly used models of cardiac gene therapy.

Indexed as

DependovirusGenetic TherapyPromoter Regions, GeneticAnimalsGenetic VectorsHumansInduced Pluripotent Stem CellsMiceMyocytes, CardiacRatsSheepSwineTroponin TTroponin T

Identifiers

PMID40683992
PMCPMC13056562

What OpenQuestion holds

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LicenceCC BY
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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.