State of the art and perspectives of gene therapy in heart failure: a scientific statement of the Heart Failure Association of the ESC, the ESC Council on Cardiovascular Genomics and the ESC Working Group on Myocardial & Pericardial Diseases
Gene therapy has recently become a reality in the treatment of cardiovascular diseases. Strategies to modulate gene expression using antisense oligonucleotides or small interfering RNA are proving to be safe and effective in the clinic. Adeno-associated viral vector-based gene delivery and CRISPR-Ca...
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| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Format: | Article (Journal) |
| Language: | English |
| Published: |
January 2025
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| In: |
European journal of heart failure
Year: 2025, Volume: 27, Issue: 1, Pages: 5-25 |
| ISSN: | 1879-0844 |
| DOI: | 10.1002/ejhf.3516 |
| Online Access: | Verlag, kostenfrei, Volltext: https://doi.org/10.1002/ejhf.3516 Verlag, kostenfrei, Volltext: http://onlinelibrary.wiley.com/doi/abs/10.1002/ejhf.3516 
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| Author Notes: | Sophie Van Linthout, Konstantinos Stellos, Mauro Giacca, Edoardo Bertero, Antonio Cannata, Lucie Carrier, Pablo Garcia-Pavia, Alessandra Ghigo, Arantxa González, Kristina H. Haugaa, Massimo Imazio, Luis R. Lopes, Patrick Most, Piero Pollesello, Heribert Schunkert, Katrin Streckfuss-Bömeke, Thomas Thum, Carlo Gabriele Tocchetti, Carsten Tschöpe, Peter van der Meer, Eva van Rooij, Marco Metra, Giuseppe M.C. Rosano, and Stephane Heymans |
MARC
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| 520 | |a Gene therapy has recently become a reality in the treatment of cardiovascular diseases. Strategies to modulate gene expression using antisense oligonucleotides or small interfering RNA are proving to be safe and effective in the clinic. Adeno-associated viral vector-based gene delivery and CRISPR-Cas9-based genome editing have emerged as efficient strategies for gene delivery and repair in humans. Overall, gene therapy holds the promise not only of expanding current treatment options, but also of intervening in previously untackled causal disease mechanisms with little side effects. This scientific statement provides a comprehensive overview of the various modalities of gene therapy used to treat heart failure and some of its risk factors, and their application in the clinical setting. It discusses specifically the possibilities of gene therapy for hereditary heart diseases and (non)-genetic heart failure. Furthermore, it addresses safety and clinical trial design issues and challenges for future regulatory strategies. | ||
| 650 | 4 | |a Adeno-associated viral vector | |
| 650 | 4 | |a Gene editing | |
| 650 | 4 | |a Gene replacement | |
| 650 | 4 | |a Gene silencing | |
| 650 | 4 | |a Gene therapy | |
| 650 | 4 | |a Heart failure | |
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