Evidence map›Paper›PMID 37674904›Full record

ArticleMolecular therapy. Methods & clinical development2023

Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.

Cindy Y Kok, Shinya Tsurusaki, Marti Cabanes-Creus, Sindhu Igoor, Renuka Rao, Rhys Skelton, Sophia H Y Liao, Samantha L Ginn, Maddison Knight, Suzanne Scott and 11 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing 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

15 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Biologics for cardiovascular diseases: from bench to bedside.Signal transduction and targeted therapy · 2026
    Review
  5. Article
  6. Review
  7. Article
  8. Article
  9. Review
  10. Article
  11. Review
  12. Review
  13. Unlocking the dark matter: noncoding RNAs and RNA modifications in cardiac aging.American journal of physiology. Heart and circulatory physiology · 2024
    Review
  14. The hunt for novel AAV capsids with improved cardiac tropism.Molecular therapy. Methods & clinical development · 2023
    Article
  15. Article
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

21 authors.

Cindy Y KokCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Shinya TsurusakiCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Marti Cabanes-CreusTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Sindhu IgoorCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Renuka RaoCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Rhys SkeltonCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Sophia H Y LiaoTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Samantha L GinnGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospital Network, Westmead, NSW 2145, Australia.
Maddison KnightTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Suzanne ScottTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Mario MietzschDepartment of Biochemistry and Molecular Biology, College of Medicine, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610-0245, USA.
Rebecca FitzsimmonsQIMR Berghofer Medical Research Institute, Herston, Brisbane, QLD 4006, Australia.
Jessica MillerInstitute of Molecular Cardiology, Department of Medicine, University of Louisville, Louisville, KY 40202, USA.
Tamer M A MohamedInstitute of Molecular Cardiology, Department of Medicine, University of Louisville, Louisville, KY 40202, USA.
Robert McKennaDepartment of Biochemistry and Molecular Biology, College of Medicine, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610-0245, USA.
James J H ChongCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.
Adam P HillVictor Chang Cardiac Research Institute, Darlinghurst, NSW 2010, Australia.
James E HudsonQIMR Berghofer Medical Research Institute, Herston, Brisbane, QLD 4006, Australia.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney and Sydney Children's Hospital Network, Westmead, NSW 2145, Australia.
Leszek LisowskiTranslational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, NSW 2145, Australia.
Eddy KizanaCentre for Heart Research, The Westmead Institute for Medical Research, The University of Sydney, Westmead, NSW 2145, Australia.

Funding

Structural studies of AAV capsids and their glycan receptor interactionsR01GM082946 · NIGMS · UNIVERSITY OF FLORIDA · PI ROBERT MCKENNA · 2007 to 2026
$6.1M
Induction of Cardiomyocyte Proliferation via Transient Expression of Cell Cycle Factors as a Promising Therapy for Heart FailureR01HL147921 · NHLBI · UNIVERSITY OF LOUISVILLE · PI Tamer M A Mohamed, Jonathan Satin · 2020 to 2026
$5.3M
Mechanisms of L-type Calcium Channel Regulation in Heart Health and DiseaseR01HL166280 · NHLBI · UNIVERSITY OF KENTUCKY · PI Jonathan Satin · 2023 to 2026
$2.5M
Physiologically relevant cardiac tissue culture model for drug testing and disease modelingR15HL168688 · NHLBI · UNIVERSITY OF LOUISVILLE · PI GIRIDHARAN, GURUPRASAD A · 2023 to 2023
$464k
Deciphering the Relationship Between Cardiomyocyte Metabolic Configuration and Cell Cycle Re-entryF32HL149140 · NHLBI · UNIVERSITY OF LOUISVILLE · PI ABOULEISA, RIHAM RAAFAT ELGHARIB · 2020 to 2023
$243k
NHLBI NIH HHS F32 HL149140NHLBI NIH HHS R01 HL147921NHLBI NIH HHS R01 HL166280NHLBI NIH HHS R15 HL168688NIGMS NIH HHS R01 GM082946
6 · The paper itself

Abstract

Recombinant adeno-associated viruses (rAAVs) have emerged as one of the most promising gene therapy vectors that have been successfully used in pre-clinical models of heart disease. However, this has not translated well to humans due to species differences in rAAV transduction efficiency. As a result, the search for human cardiotropic capsids is a major contemporary challenge. We used a capsid-shuffled rAAV library to perform directed evolution in human iPSC-derived cardiomyocytes (hiPSC-CMs). Five candidates emerged, with four presenting high sequence identity to AAV6, while a fifth divergent variant was related to AAV3b. Functional analysis of the variants was performed

Indexed as

adeno-associated viruscardiac organoidscardiac slicescardiotropichiPSC-CM

Identifiers

PMID37674904
PMCPMC10477751

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.