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Cardiac AAV9-S100A1 Gene Therapy Rescues Post-Ischemic Heart Failure in a Preclinical Large Animal Model
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A protein that regulates cellular calcium, delivered percutaneously to the heart by gene therapy, improves cardiac function in pigs with heart failure and may also be effective in humans.
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“Successful cardiac transduction in large species requires surgical and catheter-based approaches that can preferentially target the heart”
From Small and large animal models in cardiac contraction research: advantages and disadvantages (2014) · cites this paper as Pleger, et al., 2011
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“Heart failure can be induced in swine by a variety of methods such as rapid pacing (Lacroix, et al., 2002) and myocardial infarction (Pleger, et al., 2011).”
From Small and large animal models in cardiac contraction research: advantages and disadvantages (2014) · cites this paper as Pleger, et al., 2011
In the citing paper
“Myocardial gene deliveries have been performed with the aid of AAVs with methods that deliver the vectors preferentially to the myocardium such as myocardial injections (H. Su, et al., 2008), direct intracoronary (Kaspar, et al., 2005), and selective retroinfusion coupled with coronary artery occlusion (Pleger, et al., 2011; Raake, et al., 2008).”
From Small and large animal models in cardiac contraction research: advantages and disadvantages (2014) · cites this paper as Pleger, et al., 2011
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Cao, W., Chang, Y.-F., Zhao, A., Chen, B., Liu, F., Ma, Y., & Ma, X. (2017) · Journal of Molecular Histology · doi:10.1007/s10735-017-9725-4 · Cited by 2
LaRocca, T. J., & Hajjar, R. J. (2013) · Springer eBooks · doi:10.1007/978-1-4614-6705-2_44 · Cited by 2
Lyu, D., Yan, H., Chen, L., Zhang, L., Du, Y., Ding, L., & Lu, Q. (2021) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2020.593955 · Cited by 2
Knockout of Sorbs2 in Cardiomyocytes Leads to Dilated Cardiomyopathy in MicePreprint
McLendon, J. M., Zhang, X., Matasic, D. S., Kumar, M., Koval, O. M., Grumbach, I. M., Sadayappan, S., London, B., & Boudreau, R. L. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.02.13.480093 · Cited by 2
Gene Therapy for Heart Failure
Wang, H., Määttä, A., & Yla-Herttuala, S. (2026) · Journal of Cardiovascular Translational Research · doi:10.1007/s12265-025-10736-6 · Cited by 1
Gene Therapy for Cardiovascular Diseases
Weber, T., Zangi, L., & Hajjar, R. J. (2015) · Elsevier eBooks · doi:10.1016/b978-0-12-801888-0.00029-1 · Cited by 1
Gene- and Cell-Based Therapy for Cardiovascular Disease
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Gene Therapy Approaches for Disc Regeneration
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Hayward, C., Patel, H. C., Welch, S., Patel, K., & Lyon, A. R. (2017) · Encyclopedia of Life Sciences · doi:10.1002/9780470015902.a0025274 · Cited by 1
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Cellular mechanisms of diastolic dysfunction in the heart failure
Mackiewicz, U., Kołodziejczyk-Czepas, J., & Lewartowski, B. (2014) · Postępy Nauk Medycznych
Metadata
- Authors
- Pleger, S. T., Shan, C., Ksienzyk, J., Bekeredjian, R., Boekstegers, P., Hinkel, R., Schinkel, S., Leuchs, B., Ludwig, J., Qiu, G., Weber, C., Raake, P., Koch, W. J., Katus, H. A., Müller, O. J., & Most, P.
- Affiliations
- Heidelberg University (DE)
- LMU Klinikum (DE)
- German Cancer Research Center (DE)
- Thomas Jefferson University (US)
- Corresponding author
- Müller, Oliver J.
- Most, Patrick
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2011-07-20
- Volume
- 3
- Issue
- 92
- Pages
- 92ra64-92ra64
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC4095769
- OpenAlex
- W2110557028
- Cited by
- 234
- References
- 49
- Citations by year
- 2026: 5 · 2025: 10 · 2024: 10 · 2023: 10 · 2022: 14 · 2021: 15 · 2020: 17 · 2019: 16 · 2018: 14 · 2017: 16 · 2016: 19 · 2015: 22 · 2014: 15 · 2013: 22 · 2012: 24
- FWCI
- 12.97
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- OPUS (Augsburg University) (submittedVersion)
- Funders
- National Heart, Lung, and Blood Institute (R01 HL 56205, R01HL061690, R01HL92130-02S1, R01HL056205, R01 HL92130, R01HL092130, P01HL075443)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Cardiac electrophysiology and arrhythmias
- Health Sciences › Medicine › Cardiology and Cardiovascular Medicine › Cardiac Fibrosis and Remodeling
- Keywords
- S100A1, AAV9 gene therapy, cardiac gene therapy, heart failure, myocardial infarction, coronary artery occlusion, sarcoplasmic reticulum calcium handling, cardiac contractility, left ventricular remodeling, energy homeostasis, domestic pigs
- MeSH terms
- Dependovirus, Animals, Calcium, Disease Models, Animal, Energy Metabolism, Heart Failure, Heart Function Tests, Homeostasis, Humans, Myocardial Infarction, Myocardium, S100 Proteins, Organ Specificity, Sarcoplasmic Reticulum, Genetic Therapy, Biomarkers, Myocardial Ischemia, Gene Transfer Techniques, Myocytes, Cardiac, Sus scrofa
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