Evidence map›Paper›PMID 26239654›Full record

ReviewCardiovascular research2015

Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation.

Melvin Y Rincon, Thierry VandenDriessche, Marinee K Chuah

Abstract readReview
In one paragraph

Review in Cardiovascular research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 76 papers.

0numbers the graph read from it
0cells of the map it votes in
76citing 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

76 citing papers in PubMed.

  1. Review
  2. Article
  3. Gene Therapy for Heart Failure.Journal of cardiovascular translational research · 2026
    Review
  4. Frontiers in immunology · 2026
    Review
  5. Review
  6. Article
  7. Observational
  8. Review
  9. Review
  10. Article
  11. Article
  12. Article
  13. Genomic medicine and personalized treatment: a narrative review.Annals of medicine and surgery (2012) · 2025
    Review
  14. Review
  15. Article
  16. Review
  17. Article
  18. Review
  19. Cardiac gene delivery using ultrasound: State of the field.Molecular therapy. Methods & clinical development · 2024
    Review
  20. Review

16 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

3 authors.

Melvin Y RinconDepartment of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Building D, room D306, Laarbeeklaan 103, Brussels, Belgium Centro de Investigaciones, Fundacion Cardiovascular de Colombia, Floridablanca, Colombia.
Thierry VandenDriesscheDepartment of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Building D, room D306, Laarbeeklaan 103, Brussels, Belgium Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven, Belgium marinee.chuah@vub.ac.be thierry.vandendriessche@vub.ac.be.
Marinee K ChuahDepartment of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Building D, room D306, Laarbeeklaan 103, Brussels, Belgium Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven, Belgium marinee.chuah@vub.ac.be thierry.vandendriessche@vub.ac.be.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapy is a promising modality for the treatment of inherited and acquired cardiovascular diseases. The identification of the molecular pathways involved in the pathophysiology of heart failure and other associated cardiac diseases led to encouraging preclinical gene therapy studies in small and large animal models. However, the initial clinical results yielded only modest or no improvement in clinical endpoints. The presence of neutralizing antibodies and cellular immune responses directed against the viral vector and/or the gene-modified cells, the insufficient gene expression levels, and the limited gene transduction efficiencies accounted for the overall limited clinical improvements. Nevertheless, further improvements of the gene delivery technology and a better understanding of the underlying biology fostered renewed interest in gene therapy for heart failure. In particular, improved vectors based on emerging cardiotropic serotypes of the adeno-associated viral vector (AAV) are particularly well suited to coax expression of therapeutic genes in the heart. This led to new clinical trials based on the delivery of the sarcoplasmic reticulum Ca(2+)-ATPase protein (SERCA2a). Though the first clinical results were encouraging, a recent Phase IIb trial did not confirm the beneficial clinical outcomes that were initially reported. New approaches based on S100A1 and adenylate cyclase 6 are also being considered for clinical applications. Emerging paradigms based on the use of miRNA regulation or CRISPR/Cas9-based genome engineering open new therapeutic perspectives for treating cardiovascular diseases by gene therapy. Nevertheless, the continuous improvement of cardiac gene delivery is needed to allow the use of safer and more effective vector doses, ultimately bringing gene therapy for heart failure one step closer to reality.

Indexed as

Genetic TherapyGenetic VectorsGene Transfer TechniquesAdenoviridaeAnimalsCalciumCardiovascular DiseasesClinical Trials as TopicDependovirusHumansLentivirusMyocardial ContractionS100 ProteinsSarcoplasmic Reticulum Calcium-Transporting ATPasesCalciumS100A1 proteinS100 ProteinsSarcoplasmic Reticulum Calcium-Transporting ATPasesAdeno-associated viral vectorAdenylate cyclaseCardiovascular diseaseClinical trialsCRISPRGene therapyHeart failuremiRNAS100A1SERCA2a

Identifiers

PMID26239654
PMCPMC4571836

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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.